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	<title>Creative Biolabs Gene Therapy Blog</title>
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	<title>Creative Biolabs Gene Therapy Blog</title>
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		<title>Breakthrough AAV Vector for Effective Brain-Wide Gene Delivery: Targeting Human Transferrin Receptor to Enhance CNS Gene Therapy</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/breakthrough-aav-vector-for-effective-brain-wide-gene-delivery-targeting-human-transferrin-receptor-to-enhance-cns-gene-therapy/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Mon, 10 Jun 2024 08:41:43 +0000</pubDate>
				<category><![CDATA[Uncategorized]]></category>
		<category><![CDATA[AAV Vector]]></category>
		<category><![CDATA[Gene Delivery]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=217</guid>

					<description><![CDATA[The team led by Bejamine Deverman at the Broad Institute of MIT and Harvard published a research paper in the journal Science titled &#8220;An AAV capsid reprogrammed to bind human transferrin receptor<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/breakthrough-aav-vector-for-effective-brain-wide-gene-delivery-targeting-human-transferrin-receptor-to-enhance-cns-gene-therapy/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">The team led by Bejamine Deverman at the Broad Institute of MIT and Harvard published a research paper in the journal Science titled &#8220;An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery.&#8221; This study developed a novel AAV gene therapy delivery vector—BI-hTFR1—that effectively crosses the blood-brain barrier by binding to the human transferrin receptor (hTfR1), which is highly expressed in the human blood-brain barrier. When this AAV was injected into the blood of humanized mice expressing the human transferrin receptor (TfR1), the levels that entered the brain were 40-50 times higher than AAV9, which is U.S. Food and Drug Administration-approved for central nervous system gene therapy.</span></p>
<p><img decoding="async" fetchpriority="high" class="aligncenter  wp-image-218" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-scaled.jpg" alt="" width="528" height="352" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-scaled.jpg 2560w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-300x200.jpg 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-1024x683.jpg 1024w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-768x512.jpg 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-1536x1024.jpg 1536w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_314781926-2048x1365.jpg 2048w" sizes="(max-width: 528px) 100vw, 528px" /></p>
<p><span style="font-size: 15px;">Since this AAV vector functions by binding to the well-studied TfR1 protein in the blood-brain barrier, it is likely to be effective in human patients as well, laying the foundation for more effective gene therapy for central nervous system diseases.</span></p>
<p><span style="font-size: 15px;">Professor Bejamine Deverman, the corresponding author of the paper, stated, &#8220;Since establishing the laboratory at the Broad Institute in 2018, our mission has been to provide gene therapy for the central nervous system. If this new AAV works in humans as it did in our mouse studies, it will be much more effective than existing AAV vectors.&#8221;</span></p>
<p><span style="font-size: 15px;">Over the years, researchers worldwide have developed extensive AAV libraries for specific applications and tested them in animals to identify optimal candidate AAVs. However, even if this method succeeds, the selected candidate AAVs often do not work in other species, and this approach does not provide information on how AAVs reach target tissues or cells, making it challenging to translate AAV-based gene therapies from animals to humans.</span></p>
<p><span style="font-size: 15px;">To find a delivery vehicle more likely to reach the human brain, the research team turned to another approach: screening AAV libraries for AAVs that bind to specific human proteins in vitro. They then tested the most promising candidates in cells and mice that were modified to express these proteins.</span></p>
<p><span style="font-size: 15px;">In this study, they targeted the human transferrin receptor (hTfR1), which is highly expressed in the human blood-brain barrier and has long been a target for antibody therapies aimed at reaching the brain. The research team screened and identified an AAV capsid named BI-hTfR1 that binds to hTfR1, allowing it to enter human brain cells and bypass the blood-brain barrier in human cell models.</span></p>
<p><span style="font-size: 15px;">Qin Huang, the first author of the paper, developed the screening method used in this study. She said that previous in vivo screening methods have been effective but have difficulty identifying AAVs that work well across different species. This study made significant progress by finding an AAV that binds to a specific human receptor.</span></p>
<p><span style="font-size: 15px;">Professor Bejamine Deverman leads the Vector Engineering team at the Broad Institute, which is dedicated to developing innovative gene delivery solutions for central nervous system (CNS) research, using protein engineering, high-throughput in vivo selection and screening methods, and machine learning to develop new AAV vectors. Previously, Bejamine Deverman and collaborators developed AAV-PHP.B and AAV-PHP.eB, which can cross the blood-brain barrier in mice and have been widely used in laboratories worldwide.</span></p>
<p><span style="font-size: 15px;">Dr. Qin Huang is a Research Scientist on the Vector Engineering team at the Broad Institute. She graduated with a bachelor&#8217;s degree from Wuhan University, earned her Ph.D. from the University of the Chinese Academy of Sciences, and completed postdoctoral research at the University of Iowa before joining the Broad Institute in 2018.</span></p>
<p><span style="font-size: 15px;">Next, the research team tested the effectiveness of this new AAV in vivo. They created a humanized mouse model in which the transferrin receptor was replaced with human TfR1 (hTfR1), and then injected this new AAV into the blood of adult humanized mice. The results showed that, compared to mice that do not express hTfR1, the levels of AAV in the brain and spinal cord of hTfR1-expressing humanized mice were significantly higher, indicating that hTfR1 actively transports AAV across the blood-brain barrier.</span></p>
<p><span style="font-size: 15px;">The accumulation level of this new AAV in the brains of humanized mice was 40-50 times higher than that of AAV9, the vector approved for treating spinal muscular atrophy (SMA) in infants, which has lower efficiency in delivering genes to the adult brain.</span></p>
<p><span style="font-size: 15px;">Furthermore, this new AAV vector reached a large proportion of important brain cell types, targeting 71% of neurons and 92% of astrocytes.</span></p>
<p><span style="font-size: 15px;">The research team then used this new AAV vector to deliver the human GBA1 gene, which mutates in several neurological disorders associated with Gaucher disease, Lewy body dementia, and Parkinson&#8217;s disease. The results showed that the number of GBA1 gene copies delivered by this new AAV was 30 times that of AAV9 and reached most cells throughout the brain.</span></p>
<p><span style="font-size: 15px;">The research team added that this new AAV vector could be a better treatment option for neurological disorders caused by single-gene mutations (e.g., Rett syndrome, SHANK3-deficient autism), lysosomal storage diseases like GBA1 deficiency, and neurodegenerative diseases such as Huntington&#8217;s disease, prion diseases, Friedreich&#8217;s ataxia, monogenic amyotrophic lateral sclerosis, and Parkinson&#8217;s disease.</span></p>
<h5><strong>What We Do</strong></h5>
<p><span style="font-size: 15px;">Creative Biolabs provides comprehensive viral vectors and cutting-edge viral vector technology for basic research and preclinical applications, including the design and construction of suitable viral vectors and small to large-scale production of viral vectors. Our custom viral vector production stands at the forefront of gene delivery system discovery, offering a varied range of vectors that are optimized to align your project specifics while ensuring high transduction efficiency, specificity, and safety.</span><br />
<span style="font-size: 15px;">•<a href="/gene-therapy/lentiviral-vector.htm" target="_blank" rel="noopener"> Lentivirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adenovirus-vector.htm" target="_blank" rel="noopener">Adenovirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adeno-associated-virus-vector.htm" target="_blank" rel="noopener">Adeno-Associated Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/herpes-simplex-virus-vector.htm" target="_blank" rel="noopener">Herpes Simplex Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/vaccinia-viral-vector.htm" target="_blank" rel="noopener">Vaccinia Viral Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/baculovirus-vector.htm" target="_blank" rel="noopener">Baculovirus Vector</a></span><br />
<span style="font-size: 15px;">• Alphavirus Vector</span><br />
<span style="font-size: 15px;">• Flavivirus Vector</span><br />
<span style="font-size: 15px;">• Measles Virus Vector</span><br />
<span style="font-size: 15px;">• Foamy Virus Vector</span><br />
<span style="font-size: 15px;">• Hybrid Adenoviral Vector</span><br />
<span style="font-size: 15px;">• Helper-Dependent Adenoviral Vector</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Huang, Qin, et al. &#8220;An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery.&#8221; Science 384.6701 (2024): 1220-1227.</span></p>
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		<title>Breakthrough in ALS Treatment: Tofersen Significantly Slows Disease Progression in SOD1 Mutation Patient</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/breakthrough-in-als-treatment-tofersen-significantly-slows-disease-progression-in-sod1-mutation-patient/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Mon, 20 May 2024 07:32:25 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Amyotrophic Lateral Sclerosis]]></category>
		<category><![CDATA[Antisense Oligonucleotide]]></category>
		<category><![CDATA[SOD1]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=214</guid>

					<description><![CDATA[Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig&#8217;s disease, is recognized by the World Health Organization (WHO) as one of the five major incurable diseases. It is the most common motor<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/breakthrough-in-als-treatment-tofersen-significantly-slows-disease-progression-in-sod1-mutation-patient/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig&#8217;s disease, is recognized by the World Health Organization (WHO) as one of the five major incurable diseases. It is the most common motor neuron disease, and patients typically become paralyzed or die within 3-5 years of onset.</span></p>
<p><img decoding="async" class="aligncenter  wp-image-215" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-scaled.jpg" alt="" width="618" height="348" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-scaled.jpg 2560w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-300x169.jpg 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-1024x576.jpg 1024w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-768x432.jpg 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-1536x864.jpg 1536w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-2048x1152.jpg 2048w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/shutterstock_1974870962-1600x900.jpg 1600w" sizes="(max-width: 618px) 100vw, 618px" /></p>
<p><span style="font-size: 15px;">On April 25, 2023, the U.S. Food and Drug Administration approved an antisense oligonucleotide (ASO) drug called Tofersen for the treatment of ALS in adult patients with SOD1 gene mutations. This drug is administered via intrathecal injection, targeting the mRNA of the mutated SOD1 gene to promote its degradation, thereby reducing the levels of the mutated SOD1 protein and neurofilament light chain (NfL).</span></p>
<p><span style="font-size: 15px;">Recently, Umeå University in Sweden announced a breakthrough in ALS treatment. Their research team used gene therapy Tofersen to significantly slow the disease progression in an ALS patient. After four years of treatment, the patient can still climb stairs, stand up from a chair, eat, and speak well, leading an active and fulfilling social life.</span></p>
<p><span style="font-size: 15px;">Peter Andersen, the lead researcher at Umeå University, stated that this is a breakthrough in over 30 years of research, as they had never seen such effective results with any other treatment. When the patient was diagnosed in early 2020, his expected survival was only 1.5-2 years, but his response to treatment has far exceeded expectations. Tofersen has not only significantly reduced the levels of the pathogenic SOD1 protein but also significantly inhibited the disease progression.</span></p>
<p><span style="font-size: 15px;">The patient, from a family in southern Sweden, had a relative diagnosed with ALS, and his blood sample was provided to the ALS research team at Umeå University. However, he chose not to be informed of the genetic test results at that time. In fact, he did carry the pathogenic gene mutation for ALS. Four years ago, after showing symptoms of muscle weakness, he was officially diagnosed with ALS caused by the SOD1 gene mutation.</span></p>
<p><span style="font-size: 15px;">Since the summer of 2020, the patient has been participating in a Phase 3 clinical trial of Tofersen for ALS with SOD1 gene mutations. This trial targets ALS patients with SOD1 gene mutations that cause the misfolding and aggregation of the SOD1 protein in motor neurons. Patients receive experimental treatment once every four weeks.</span></p>
<p><span style="font-size: 15px;">When a person’s SOD1 gene is mutated, it produces a mutated SOD1 protein in the brain and spinal cord. Over time, the accumulation of this protein kills nerve cells that control muscle movement, leading to ALS symptoms. Damaged or destroyed nerve cells release neurofilaments; the more mutated SOD1 protein accumulates, the more nerve cells are killed, increasing neurofilament levels in cerebrospinal fluid and blood. Thus, neurofilament levels are considered an important biomarker for ALS disease progression.</span></p>
<p><span style="font-size: 15px;">When the patient was diagnosed in April 2020, his ALS biomarker neurofilament light chain (NfL) levels were very high, reaching 11,000 ng/L in cerebrospinal fluid, which is high even among ALS patients. Four years later, his NfL levels have dropped to around 1,200 ng/L, a nearly 90% reduction. In healthy individuals of his age group, NfL levels in cerebrospinal fluid are typically below 560 ng/L. His blood NfL levels have returned to normal, with the latest measurement at 12 ng/L, within the normal range of below 13 ng/L.</span></p>
<p><span style="font-size: 15px;">The ALSFRS-R scale shows that the patient&#8217;s motor function level, although lower than that of a healthy person (48 points), has remained stable at around 35-37 points over the past 18 months, indicating a decline of about 26% compared to healthy individuals.</span></p>
<p><span style="font-size: 15px;">For patients with aggressive ALS caused by SOD1 gene mutations, their ALSFRS-R scores usually drop by 1-1.5 points per month. Without treatment, the patient’s disease progression would have been very rapid, likely leading to severe disability within 6-12 months and death within 1.5-2 years. Now, four years after onset, the patient can still climb stairs, which is miraculous. His life has not been significantly impacted; he speaks normally, shops, takes care of his children, and feels mentally much better, regaining hope in life.</span></p>
<p><span style="font-size: 15px;">ALS is a complex disease, with only 2%-6% of patients having SOD1 gene mutations, which are usually familial. However, some sporadic ALS patients also have SOD1 gene mutations. It is currently unclear whether Tofersen has similar effects on other types of ALS, requiring further exploration.</span></p>
<p><span style="font-size: 15px;">Tofersen has been approved by the U.S. Food and Drug Administration and the European Medicines Agency (EMA) for treating adult ALS patients with SOD1 gene mutations.</span></p>
<p><span style="font-size: 15px;">The research team stated that the next step is to study the outcomes of patients treated with Tofersen. While effective for some patients, it has not produced the same positive results for all, which might be due to dosage issues or treatment at different stages of ALS, all of which need further investigation.</span></p>
<h5><strong>What We Do</strong></h5>
<p><span style="font-size: 15px;">As one of the leading biotechnology companies in the world, Creative Biolabs has sophisticated equipment and highly experienced staff. Scientists at Creative Biolabs are able to provide you with <span style="color: #0000ff;"><a style="color: #0000ff;" href="/gene-therapy/custom-antisense-oligonucleotide-synthesis.htm" target="_blank" rel="noopener"><strong>custom antisense oligonucleotide synthesis</strong></a></span> services. We also offer a series of ready-to-use <span style="color: #0000ff;"><strong><a style="color: #0000ff;" href="/gene-therapy/antisense-oligonucleotides-asos.htm" target="_blank" rel="noopener">antisense oligonucleotide</a></strong></span> products for various disease research, including cancer, cytomegalovirus retinitis, familial hypercholesterolemia, viral hemorrhagic fever, HIV/AIDS, spinal muscular atrophy, Duchenne muscular, hypertriglyceridemia and other human diseases. Please feel free to contact us to learn more about our capabilities and products.</span></p>
<p>&nbsp;</p>
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		<title>Advancements in Gene Therapy: Ring Therapeutics Develops Anellovirus-Based Anellovector for Safe and Effective Treatment</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/advancements-in-gene-therapy-ring-therapeutics-develops-anellovirus-based-anellovector-for-safe-and-effective-treatment/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Wed, 10 Apr 2024 07:21:58 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Anellovirus]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=211</guid>

					<description><![CDATA[Over the past few decades, viruses have been transformed into life-saving therapies, including for vaccine development and cancer treatment. The latest advancement in using viruses to treat human diseases is gene therapy—using<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/advancements-in-gene-therapy-ring-therapeutics-develops-anellovirus-based-anellovector-for-safe-and-effective-treatment/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">Over the past few decades, viruses have been transformed into life-saving therapies, including for vaccine development and cancer treatment. The latest advancement in using viruses to treat human diseases is gene therapy—using engineered viruses as delivery vectors to replace or repair genetic defects.</span></p>
<p><span style="font-size: 15px;">Although these virus-based therapies and vaccines are game-changers, they also face numerous obstacles, especially the immunogenicity of viruses, which limits their therapeutic potential. For example, the FDA has approved several in vivo gene therapies based on adeno-associated virus (AAV), which have shown safe and effective treatment for genetic diseases. However, up to 70% of humans already have antibodies against AAV, and those without antibodies will develop them after receiving AAV gene therapy. This means AAV gene therapies can only be used once, requiring a sufficiently high dose to achieve enough therapeutic effect in one go, which significantly increases side effects at high doses. Lentivirus (LV)-based delivery vectors, on the other hand, integrate the delivered sequences into the human genome, posing a potential risk of carcinogenesis.</span></p>
<p><span style="font-size: 15px;">If we could utilize naturally occurring viruses in the human body as delivery vectors, it could address current gene therapy issues like safety risks, inability to administer repeat doses, and poor tolerance.</span></p>
<p><span style="font-size: 15px;">Ring Therapeutics, a biotechnology company incubated by the renowned venture capital firm Flagship Pioneering, has raised over $250 million, with its latest $86.5 million Series C round completed in March 2023.</span></p>
<p><span style="font-size: 15px;">Researchers at Ring discovered a diverse family of viruses called anelloviruses, which make up a significant portion of the human commensal virome. Anelloviruses have co-evolved with humans for thousands of years, stably residing within cells of various human tissues without triggering the immune system. The company aims to develop a programmable delivery platform based on anelloviruses to treat various human diseases more safely and effectively.</span></p>
<p><span style="font-size: 15px;">On March 30, 2024, Ring’s researchers published a study on the preprint platform bioRxiv titled: &#8220;A novel functional gene delivery platform based on a commensal human anellovirus demonstrates transduction in multiple tissue types.&#8221;</span></p>
<p><img decoding="async" class="aligncenter  wp-image-212" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/457475.png" alt="" width="545" height="359" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/457475.png 1533w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/457475-300x198.png 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/457475-1024x675.png 1024w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/07/457475-768x506.png 768w" sizes="(max-width: 545px) 100vw, 545px" /></p>
<p><span style="font-size: 15px;">This study reported the first gene delivery vector system based on the human commensal virus—Anellovector. Anellovector showed sustained expression in mouse eyes for nine months after subretinal injection and comparable expression levels to AAV9 in the brain after intracerebroventricular injection. The newly developed viral delivery vector Anellovector has great potential to provide safe, repeatable, and effective treatments, expanding the application scope of programmable gene therapies.</span></p>
<p><span style="font-size: 15px;">Anelloviruses are non-enveloped viruses with a negative-sense, circular, single-stranded DNA genome that can infect vertebrates and are a prevalent component of the human commensal virome. Human anelloviruses can evade the humoral immune response and appear to be non-pathogenic (no association with any human disease has been identified to date). These characteristics, combined with their extensive genomic diversity and widespread tissue distribution in humans, make anelloviruses strong candidates for next-generation gene drug delivery vectors.</span></p>
<p><span style="font-size: 15px;">Ring Therapeutics is searching for new human viruses that allow multiple gene therapy treatments within a single patient, and new mouse data suggests they may have found a viable candidate.</span></p>
<p><span style="font-size: 15px;">The research team developed a new viral vector based on Ring&#8217;s proprietary human anellovirus Anellogy platform—Anellovector. Using this vector, they successfully delivered the gene for green fluorescent protein (GFP) into mouse retinas, showing stable expression for up to nine months without any signs of toxicity.</span></p>
<p><span style="font-size: 15px;">Dr. Tuyen Ong, CEO of Ring Therapeutics, stated that they have rapidly achieved the development of a novel delivery vector system based on human commensal viruses to address many challenges faced by current gene drug delivery. This latest study shows that they can successfully utilize the properties of anelloviruses to generate the viral vector—Anellovector, marking the first new viral vector in decades.</span></p>
<p><span style="font-size: 15px;">The company developed a new viral vector based on the Betatorquevirus genus of anelloviruses—Anellovector. In this study, the research team tested Anellovector&#8217;s ability to deliver the GFP gene to retinal cells. In living mice, Anellovector successfully delivered the GFP gene to retinal cells, and the expression levels were measured at three, six, and nine months. They found that although the delivered DNA copy number decreased over time, its expression levels remained stable between three to nine months without any signs of toxicity.</span></p>
<p><span style="font-size: 15px;">To see how Anellovector compared to AAV vectors and whether they worked in tissues outside the eye, the team injected a dose of either AAV9 (one of the most commonly used AAV vectors) or Anellovector into the left and right brain ventricles of two groups of mice. After 21 days, the expression levels of the Anellovector and AAV9 vectors were roughly equivalent. Brain slices showed similar expression levels between the two groups. These studies indicate that Anellovector has an affinity for cells in the central nervous system and its gene delivery efficiency is comparable to the commonly used AAV9 vector.</span></p>
<h5><strong>What We Do</strong></h5>
<p><span style="font-size: 15px;">Creative Biolabs provides comprehensive viral vectors and cutting-edge viral vector technology for basic research and preclinical applications, including the design and construction of suitable viral vectors and small to large-scale production of viral vectors. Our custom viral vector production stands at the forefront of gene delivery system discovery, offering a varied range of vectors that are optimized to align your project specifics while ensuring high transduction efficiency, specificity, and safety.</span><br />
<span style="font-size: 15px;">•<a href="/gene-therapy/lentiviral-vector.htm" target="_blank" rel="noopener"> Lentivirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adenovirus-vector.htm" target="_blank" rel="noopener">Adenovirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adeno-associated-virus-vector.htm" target="_blank" rel="noopener">Adeno-Associated Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/herpes-simplex-virus-vector.htm" target="_blank" rel="noopener">Herpes Simplex Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/vaccinia-viral-vector.htm" target="_blank" rel="noopener">Vaccinia Viral Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/baculovirus-vector.htm" target="_blank" rel="noopener">Baculovirus Vector</a></span><br />
<span style="font-size: 15px;">• Alphavirus Vector</span><br />
<span style="font-size: 15px;">• Flavivirus Vector</span><br />
<span style="font-size: 15px;">• Measles Virus Vector</span><br />
<span style="font-size: 15px;">• Foamy Virus Vector</span><br />
<span style="font-size: 15px;">• Hybrid Adenoviral Vector</span><br />
<span style="font-size: 15px;">• Helper-Dependent Adenoviral Vector</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Prince, Cato, et al. &#8220;A novel functional gene delivery platform based on a commensal human anellovirus demonstrates transduction in multiple tissue types.&#8221; <i>bioRxiv</i> (2024): 2024-03.</span></p>
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		<title>Revolutionizing Gene Therapy: Human Liver Model Advances Preclinical Testing</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/revolutionizing-gene-therapy-human-liver-model-advances-preclinical-testing/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Wed, 27 Mar 2024 05:59:21 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<category><![CDATA[Human Liver Model]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=198</guid>

					<description><![CDATA[Gene therapy is a revolutionary approach for treating severe genetic diseases, typically involving the replacement or repair of faulty genes. Currently, the most effective delivery system is based on adeno-associated virus vector,<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/revolutionizing-gene-therapy-human-liver-model-advances-preclinical-testing/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">Gene therapy is a revolutionary approach for treating severe genetic diseases, typically involving the replacement or repair of faulty genes. Currently, the most effective delivery system is based on <a href="/gene-therapy/adeno-associated-virus-vector.htm" target="_blank" rel="noopener"><strong><span style="color: #0000ff;">adeno-associated virus vector</span></strong></a>, which naturally possesses the ability to introduce genetic information into human cells.</span></p>
<p><span style="font-size: 15px;">One of the biggest challenges scientists face in transitioning gene therapy from the lab to the clinic is the lack of effective preclinical models available for developing and testing new therapies, such as laboratory tests with biological relevance and clinical predictability conducted before applying a therapy to patients. These models must faithfully replicate human physiological conditions and complex tissue structures to accurately predict the outcomes of treatment in patients.</span></p>
<p><span style="font-size: 15px;">Previously, scientists established a novel method for maintaining the viability of human livers in a laboratory environment. Livers unsuitable for transplantation—previously discarded or stored on ice for research—are now able to be preserved ex vivo at body temperature, enabling cutting-edge biomedical research. This is known as the normothermic liver perfusion system.</span></p>
<p><span style="font-size: 15px;">In a groundbreaking global study, researchers from institutions including the University of Sydney in Australia tested a novel gene therapy within intact human livers, aiming to develop more effective methods for treating life-threatening genetic diseases. The related research findings were published in the journal Nature Communications on March 14, 2024, titled &#8220;Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.&#8221;</span></p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-200" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML.png" alt="" width="533" height="543" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML.png 2000w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML-294x300.png 294w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML-1005x1024.png 1005w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML-768x783.png 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/03/41467_2024_46194_Fig1_HTML-1507x1536.png 1507w" sizes="(max-width: 533px) 100vw, 533px" /></p>
<p><span style="font-size: 15px;">These authors confirmed that testing AAV-based therapies using this system before initiating clinical studies is feasible. Utilizing intact human livers represents a revolutionary advancement in the field of <a href="/gene-therapy/service.htm" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene therapy</strong></span></a> because it allows for precise testing of how new therapies will impact a major organ—the liver—in a manner previously unattainable.</span></p>
<p><span style="font-size: 15px;">Associate Professor Leszek Lisowski, the corresponding author of the paper, said, &#8220;This is tremendously exciting because for the first time we can directly evaluate the function of gene therapy in a clinical target organ—the human liver.&#8221;</span></p>
<p><span style="font-size: 15px;">He added, &#8220;This is critical because the viral vectors we currently use for delivering gene therapy to the liver are not yet sufficient for most clinical applications. Currently, we often have to deliver high doses to overcome their low functionality and achieve clinical efficacy. Gene therapy delivery tools have been tested in animal models thus far, and while animal models are valuable for assessing safety and targeting other organs/tissues, they do not fully recapitulate the functionality of these delivery methods in patients.&#8221;</span></p>
<p><span style="font-size: 15px;">He further stated, &#8220;This research expands the scope of preclinical models available for liver-targeted vector research, allowing us to minimize the use of animals. Ideally, this will bring us closer to providing more effective gene therapy for diseases that currently have limited treatment options.&#8221;</span></p>
<p><span style="font-size: 15px;">Lisowski noted that this new advanced human liver model not only changes the game for functional assessment of novel therapies but also allows for more accurate estimation of effective doses of new therapies and identification of potential toxic side effects. It will also serve as a robust system for developing novel viral vectors, laying the foundation for the next generation of advanced therapies.</span></p>
<h5><strong>What We Do</strong></h5>
<p><span style="font-size: 15px;">Creative Biolabs provides comprehensive viral vectors and cutting-edge viral vector technology for basic research and preclinical applications, including the design and construction of suitable viral vectors and small to large-scale production of viral vectors. Our custom viral vector production stands at the forefront of gene delivery system discovery, offering a varied range of vectors that are optimized to align your project specifics while ensuring high transduction efficiency, specificity, and safety.</span><br />
<span style="font-size: 15px;">•<a href="/gene-therapy/lentiviral-vector.htm" target="_blank" rel="noopener"> Lentivirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adenovirus-vector.htm" target="_blank" rel="noopener">Adenovirus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/adeno-associated-virus-vector.htm" target="_blank" rel="noopener">Adeno-Associated Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/herpes-simplex-virus-vector.htm" target="_blank" rel="noopener">Herpes Simplex Virus Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/vaccinia-viral-vector.htm" target="_blank" rel="noopener">Vaccinia Viral Vector</a></span><br />
<span style="font-size: 15px;">• <a href="/gene-therapy/baculovirus-vector.htm" target="_blank" rel="noopener">Baculovirus Vector</a></span><br />
<span style="font-size: 15px;">• Alphavirus Vector</span><br />
<span style="font-size: 15px;">• Flavivirus Vector</span><br />
<span style="font-size: 15px;">• Measles Virus Vector</span><br />
<span style="font-size: 15px;">• Foamy Virus Vector</span><br />
<span style="font-size: 15px;">• Hybrid Adenoviral Vector</span><br />
<span style="font-size: 15px;">• Helper-Dependent Adenoviral Vector</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Cabanes-Creus, Marti, <em>et al</em>. &#8220;Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.&#8221; Nature Communications 15.1 (2024): 1876.</span></p>
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		<title>PRINT: Enhancing Gene Therapy with Precision Retrotransposon Insertion</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/print-enhancing-gene-therapy-with-precision-retrotransposon-insertion/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Mon, 26 Feb 2024 06:10:58 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<category><![CDATA[Precision Retrotransposon Insertion]]></category>
		<category><![CDATA[PRINT]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=202</guid>

					<description><![CDATA[Recently, a CRISPR-Cas9 therapy for sickle cell disease has been approved, showcasing the potential of gene editing tools to effectively knock out genes for treating genetic disorders. However, the replacement of entire<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/print-enhancing-gene-therapy-with-precision-retrotransposon-insertion/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">Recently, a CRISPR-Cas9 therapy for sickle cell disease has been approved, showcasing the potential of <a href="/gene-therapy/gene-editing-for-gene-therapy.htm" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene editing</strong></span></a> tools to effectively knock out genes for treating genetic disorders. However, the replacement of entire genes in the human genome to substitute defective or harmful genes remains unfeasible.</span></p>
<p><span style="font-size: 15px;">A new technology utilizing retrotransposons derived from birds to insert genes into the genome brings more hope for gene therapy. This technology allows the insertion of genes into &#8220;safe harbors&#8221; in the human genome, where inserted genes do not disrupt vital genes or cause cancer.</span></p>
<p><span style="font-size: 15px;">Retrotransposons are DNA segments that, when transcribed into RNA, encode enzymes that copy RNA into DNA in the genome—a self-serving cycle that fills the genome with retrotransposon DNA. Approximately 40% of the human genome consists of this &#8220;selfish&#8221; new DNA, though much of it has lost function, termed &#8220;junk DNA.&#8221;</span></p>
<p><span style="font-size: 15px;">This new technology, called &#8220;Precise RNA-mediated INsertion of Transgenes (PRINT),&#8221; harnesses the ability of certain retrotransposons to effectively insert entire genes into the genome without affecting other genomic functions. PRINT complements the capabilities of CRISPR-Cas technology in gene knockout, point mutations, and short DNA fragment insertion.</span></p>
<p><span style="font-size: 15px;">PRINT was developed by Professor Kathleen Collins&#8217; laboratory at the University of California, Berkeley. Described in a paper published online in the journal Nature Biotechnology on February 20, 2024, titled &#8220;Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci.&#8221;</span></p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-203" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML.png" alt="" width="590" height="570" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML.png 2164w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML-300x290.png 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML-1024x991.png 1024w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML-768x743.png 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML-1536x1486.png 1536w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/04/41587_2024_2137_Fig1_HTML-2048x1982.png 2048w" sizes="(max-width: 590px) 100vw, 590px" /></p>
<p><span style="font-size: 15px;">PRINT involves using a delivery method similar to delivering CRISPR-Cas9 into cells for genome editing to insert new DNA into cells. In PRINT, a delivered RNA encodes a common retrotransposon factor called the R2 protein, which contains multiple functional domains, including a nickase—an enzyme that binds to and cuts double-stranded DNA—and a reverse transcriptase—an enzyme that uses DNA as a template to produce RNA. Another RNA acts as a template for inserting transgenic DNA, along with gene expression control elements, forming an entire autonomous transgenic cassette in the genome.</span></p>
<p><span style="font-size: 15px;">One key advantage of using the R2 protein is its ability to insert transgenes into regions of the genome containing hundreds of copies of the same gene—each copy encoding ribosomal RNA, which is the RNA machinery translating messenger RNA (mRNA) into proteins. With so many redundant copies, disruption of one or a few ribosomal RNA genes by the inserted gene does not have adverse effects.</span></p>
<p><span style="font-size: 15px;">Inserting transgenes into safe harbors avoids a major issue encountered when inserting transgenes via viral vectors into the genome: genes are often randomly inserted, leading to loss of function in working genes or disruption of gene regulation or function, potentially resulting in cancer.</span></p>
<p><span style="font-size: 15px;">Collins stated, &#8220;CRISPR-Cas9-based methods can fix a mutated nucleotide or insert a small DNA sequence. Or you can knock out gene function through site-specific mutagenesis. We&#8217;re not knocking out gene function. We&#8217;re not fixing endogenous gene mutations. We&#8217;re taking a complementary approach by adding a gene into the genome that can autonomously express a protein, serving as a missing bypass, re-adding a functional gene. This is transgenic supplementation, not gene mutation reversal. This is a very good approach for repairing loss-of-function diseases caused by a series of individual mutations in the same gene.&#8221;</span></p>
<p><span style="font-size: 15px;">Many genetic diseases, such as cystic fibrosis and hemophilia, are caused by multiple different mutations in the same gene, all of which result in loss of gene function. Any CRISPR-Cas9-based gene editing therapy must be tailored to the specific mutations of the patient. With PRINT for gene supplementation, the correct genes can be delivered to each patient, allowing each patient&#8217;s body to produce normal proteins regardless of the original gene mutations.</span></p>
<p><span style="font-size: 15px;">Many academic labs and startups are researching how to use transposons and retrotransposons to insert genes for <a href="/gene-therapy/service.htm" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene therapy</strong></span></a>. A popular retrotransposon being studied by biotech companies is LINE-1 (Long INterspersed Element-1), which can replicate itself and some hitchhiking genes in humans, occupying about 30% of the entire genome, although fewer than 100 copies of LINE-1 retrotransposons in the human genome are functional, constituting a tiny fraction of the genome.</span></p>
<p><span style="font-size: 15px;">Collins, along with her postdoctoral colleague Akanksha Thawani and Professor Eva Nogales from the University of California, Berkeley, published the low-temperature electron microscopy structure of LINE-1-encoded enzyme protein in Nature on December 14, 2023 (Nature, 2023, doi:10.1038/s41586-023-06933-5).</span></p>
<p><span style="font-size: 15px;">Collins said that this new research clearly indicates that LINE-1 retrotransposon proteins have difficulty in effectively and safely inserting transgenes into the human genome through genetic manipulation. However, previous studies have shown that genes inserted into the repetitive ribosomal RNA coding regions (rDNA) of the genome can be expressed normally, which led Collins to consider another retrotransposon factor named R2, which might be better suited for safe gene insertion.</span></p>
<p><span style="font-size: 15px;">Since R2 does not exist in the human body, Collins and her senior researcher Xiaozhu Zhang and postdoctoral researcher Briana Van Treeck from the University of California, Berkeley, screened R2 from dozens of animal genomes ranging from insects to horseshoe crabs and other multicellular eukaryotes to find a version highly targeted to the rDNA region in the human genome and efficiently insert long DNA into that area.</span></p>
<p><span style="font-size: 15px;">Collins said, &#8220;After searching dozens of species, the real winners came from birds,&#8221; including zebra finches and white-throated sparrows. She noted that while mammals lack R2 in their genomes, they have binding sites necessary for R2 as a retrotransposon factor to insert effectively—suggesting that mammals&#8217; ancestors might have had a R2-like retrotransposon factor, which was somehow purged from mammalian genomes.</span></p>
<p><span style="font-size: 15px;">In experiments, Zhang and Van Treeck synthesized mRNA encoding the R2 protein and a template RNA producing a transgene carrying a fluorescent protein, whose expression is controlled by an RNA polymerase promoter. These were co-transfected into human cells cultured in vitro. Under laser irradiation, about half of the cells lit up green or red due to expression of the fluorescent protein, indicating successful insertion of the working fluorescent protein by the R2 system into the genome.</span></p>
<p><span style="font-size: 15px;">Further studies showed that the transgene indeed inserted into the rDNA region of the genome, and approximately 10 copies of the RNA template could be inserted without disrupting the protein-making activity of the rDNA genes.</span></p>
<p><span style="font-size: 15px;">Besides providing safe harbors, inserting transgenes into the genome&#8217;s rDNA region offers other benefits. The rDNA region is located on the short arms of five independent chromosomes, all packed together to form a structure called the nucleolus, where DNA is transcribed into ribosomal RNA and folded into the ribosomal machinery for protein synthesis.</span></p>
<p><span style="font-size: 15px;">Within the nucleolus, transcription of rDNA is highly regulated, and its genes are rapidly repaired, as any breaks in rDNA, if left unchecked, can halt protein production. Therefore, any transgenes inserted into the genome&#8217;s rDNA region are tightly protected within the nucleolus.</span></p>
<p><span style="font-size: 15px;">Collins said, &#8220;The nucleolus is a huge ribosome biogenesis center. But it&#8217;s also an extremely privileged DNA repair environment, with</span></p>
<p><span style="font-size: 15px;">a low risk of carcinogenesis from gene insertion. These successful retrotransposons entering the nucleolus DNA are really remarkable. It&#8217;s multicopy, it&#8217;s conservative, it&#8217;s a safe harbor, you can destroy one copy, and the cell doesn&#8217;t care.&#8221; This makes the region an ideal location for inserting genes for human gene therapy.</span></p>
<p><span style="font-size: 15px;">Collins acknowledges that there are still many unknowns about the mechanism of R2 and biological questions about rDNA transcription: how many rDNA genes will be disrupted before the cell takes notice? Since some cells shut down many genes from the 400+ rDNA genes in the human genome, are these cells more susceptible to the side effects of PRINT?</span></p>
<p><span style="font-size: 15px;">She and her team are researching these questions while also tweaking various proteins and RNAs involved in retrotransposon insertion to make PRINT more effective in cells cultured in vitro and primary cells from human tissues.</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Zhang, Xiaozhu, <em>et al</em>. &#8220;Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci.&#8221; Nature Biotechnology (2024): 1-10.</span></p>
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		<title>New Gene Therapy Offers Hope for Treating Devastating Childhood Epilepsy: A Study from UCL Researchers</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/new-gene-therapy-offers-hope-for-treating-devastating-childhood-epilepsy-a-study-from-ucl-researchers/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Sat, 06 Jan 2024 05:48:15 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Devastating Childhood Epilepsy]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=192</guid>

					<description><![CDATA[In a new study, researchers from University College London have developed a novel gene therapy to treat a devastating form of childhood epilepsy, showing significant reduction in seizure episodes in mouse models.<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/new-gene-therapy-offers-hope-for-treating-devastating-childhood-epilepsy-a-study-from-ucl-researchers/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p>In a new study, researchers from University College London have developed a novel gene therapy to treat a devastating form of childhood epilepsy, showing significant reduction in seizure episodes in mouse models. This discovery holds promise for providing an alternative to surgery for children suffering from focal cortical dysplasia. The findings were published in the journal Brain, with the paper titled &#8220;Anti-seizure Gene Therapy for Focal Cortical Dysplasia.&#8221;</p>
<p><img decoding="async" loading="lazy" class="aligncenter  wp-image-193" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-scaled.jpeg" alt="" width="616" height="757" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-scaled.jpeg 2084w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-244x300.jpeg 244w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-834x1024.jpeg 834w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-768x943.jpeg 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-1251x1536.jpeg 1251w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/awad387f1-1667x2048.jpeg 1667w" sizes="(max-width: 616px) 100vw, 616px" /></p>
<p>Focal cortical dysplasia is caused by regions of abnormal brain development and is one of the most common reasons behind drug-resistant epilepsy in children. It often occurs in the frontal lobes, which are crucial for planning and decision-making. Epilepsy associated with focal cortical dysplasia is linked to comorbidities, including learning disabilities. Although surgical removal of the affected brain malformation is effective, its use is severely limited by the risk of permanent neurological deficits, and it does not always prevent the recurrence of epilepsy.</p>
<p>Therefore, in this new study, the authors evaluated a <a href="/gene-therapy/service.htm" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene therapy</strong></span></a> in mouse models of frontal lobe focal cortical dysplasia. This therapy is based on the overexpression of a potassium channel that regulates neuronal excitability. Potassium channels control the flow of potassium ions in and out of cells. Overexpression of potassium channels means enhanced regulation, leading to reduced cell activity and thereby preventing seizures.</p>
<p>&#8220;We are very excited to see that this novel gene therapy has the potential to be an effective alternative to surgical treatment for patients with focal cortical dysplasia,&#8221; said Professor Gabriele Lignani, co-corresponding author and a researcher at the Queen Square Institute of Neurology, University College London.</p>
<p>Gene therapy has previously been proven effective for another form of epilepsy occurring in the temporal lobe but had not been tested in focal cortical dysplasia. In this new study, the authors introduced an engineered potassium channel gene named EKC into the affected epileptic frontal lobe of mice. To increase safety, they used a non-replicating virus to carry the potassium channel gene. Before treatment, they monitored the brain activity of the mice for 15 days. They then injected the virus carrying the EKC gene or a control virus into the affected brain area. Subsequently, they monitored the brain activity of the mice for another 15 days.</p>
<p>They found that, compared to control mice, the gene therapy reduced seizures by an average of 87% in mice that received the therapy, without affecting their memory or behavior.</p>
<p>&#8220;After the success in mouse studies, we believe this therapy is suitable for clinical translation and, considering the scale of unmet needs, it could potentially be applied to thousands of children currently severely affected by uncontrolled epileptic seizures,&#8221; said Dr. Vincent Magloire, co-author and researcher at the Queen Square Institute of Neurology, University College London.</p>
<p>Professor Dimitri Kullmann, co-corresponding author and researcher at the Queen Square Institute of Neurology, University College London, added, &#8220;Plans for the first human clinical trials are underway, with completion anticipated within the next five years.&#8221;</p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Almacellas Barbanoj, Amanda, et al. &#8220;Anti-seizure gene therapy for focal cortical dysplasia.&#8221; Brain 147.2 (2024): 542-553.</span></p>
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		<title>Gene Therapy for Arrhythmogenic Cardiomyopathy: Pioneering Research from Utrecht University Medical Center</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/gene-therapy-for-arrhythmogenic-cardiomyopathy-pioneering-research-from-utrecht-university-medical-center/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Sat, 16 Dec 2023 05:33:01 +0000</pubDate>
				<category><![CDATA[Uncategorized]]></category>
		<category><![CDATA[Adeno-associated Virus]]></category>
		<category><![CDATA[Arrhythmogenic Cardiomyopathy]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=189</guid>

					<description><![CDATA[Recently, researchers from the Utrecht University Medical Center published a research paper in the Nature subsidiary journal, Nature Cardiovascular Research, titled: &#8220;Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.&#8221; The<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/gene-therapy-for-arrhythmogenic-cardiomyopathy-pioneering-research-from-utrecht-university-medical-center/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p>Recently, researchers from the Utrecht University Medical Center published a research paper in the Nature subsidiary journal, Nature Cardiovascular Research, titled: &#8220;Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.&#8221; The study, which utilizes <a href="/gene-therapy/category-recombinant-adeno-associated-virus-304.htm" target="_blank" rel="noopener"><strong>r<span style="color: #0000ff;">ecombinant adeno-associated virus</span></strong></a> delivery of the PKP2 gene for treatment, lays the foundation for gene therapy in genetic heart diseases—specifically, arrhythmogenic cardiomyopathy (ACM). This therapy is scheduled for multiple clinical trials in the United States in 2024.</p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-190" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML.png" alt="" width="660" height="741" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML.png 1789w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML-267x300.png 267w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML-913x1024.png 913w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML-768x862.png 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2024/02/44161_2023_378_Fig1_HTML-1369x1536.png 1369w" sizes="(max-width: 660px) 100vw, 660px" /></p>
<p>Arrhythmogenic cardiomyopathy (ACM) is often caused by mutations in genes associated with desmosomes, which are responsible for connecting adjacent cardiac muscle cells. Not only do they provide structural connectivity, but they also ensure the synchronized contraction of cardiac muscle cells, allowing the heart to pump blood in a coordinated manner.</p>
<p>The most common mutation in ACM affects the PKP2 gene, which encodes for an essential component of the desmosome—plakophilin-2. Patients with this mutation typically have lower levels of plakophilin-2 protein in cardiac muscle cells, leading to weakened intercellular connections, making it difficult for them to work in sync, and resulting in the development of arrhythmias.</p>
<p>Therefore, the research team considered developing a gene therapy targeting the PKP2 gene mutation to fundamentally treat arrhythmogenic cardiomyopathy (ACM). Introducing the correct PKP2 gene into affected cardiac muscle cells could potentially restore plakophilin-2 protein levels to normal, thereby strengthening the desmosomes and reducing the incidence of arrhythmias in these patients.</p>
<p>Using several laboratory models of arrhythmogenic cardiomyopathy (ACM), the research team demonstrated that delivering the correct PKP2 gene to human cardiac muscle cells derived from stem cells restored plakophilin-2 levels and improved their sodium ion conductance, which is important for the contractile ability of cardiac muscle cells.</p>
<p>Next, the research team confirmed the therapy&#8217;s beneficial effects on cardiac muscle contractility in laboratory-cultured engineered human cardiac muscle cells. Finally, the effectiveness of PKP2 gene therapy was further validated in a mouse model of arrhythmogenic cardiomyopathy (ACM), successfully improving the desmosomes and cardiac function recovery in the mouse model.</p>
<p>Following promising preclinical research progress, the next step is to explore the clinical therapeutic potential of this gene therapy method in patients with arrhythmogenic cardiomyopathy (ACM) carrying PKP2 gene mutations.</p>
<p>Eirini Kyriakopoulou, the lead author of the paper, stated that three companies in the United States have announced that they will start clinical trials next year to test the therapeutic effects of this <a href="/gene-therapy/service.htm" target="_blank" rel="noopener"><strong><span style="color: #0000ff;">gene therapy</span></strong></a> in patients. Once arrhythmogenic cardiomyopathy (ACM) progresses to a certain extent, part of the myocardium has already been replaced by fatty tissue, and whether this method can reverse existing myocardial damage remains uncertain. However, it may be sufficient to prevent early disease progression to more severe stages.</p>
<p>Although preclinical experimental results and the upcoming clinical trials bring great hope for arrhythmogenic cardiomyopathy (ACM), the true commercialization of this therapy may still take several years. In addition to confirming its efficacy in human patients, any safety concerns must be addressed and resolved before clinical application.</p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Kyriakopoulou, Eirini, et al. &#8220;Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.&#8221; Nature Cardiovascular Research (2023): 1-15.</span></p>
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		<title>Gene Therapy Holds Promise for Paralysis Caused by CNTNAP1 Mutations</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/gene-therapy-holds-promise-for-paralysis-caused-by-cntnap1-mutations/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Fri, 03 Nov 2023 05:35:18 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[CNTNAP1]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=161</guid>

					<description><![CDATA[In 50 families from the Netherlands, the United Kingdom, the United States, and China, each family has a child paralyzed due to a mutation in the Cntnap1 (Contactin-Associated Protein 1) gene. These<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/gene-therapy-holds-promise-for-paralysis-caused-by-cntnap1-mutations/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">In 50 families from the Netherlands, the United Kingdom, the United States, and China, each family has a child paralyzed due to a mutation in the Cntnap1 (Contactin-Associated Protein 1) gene. These children are immobile, requiring assistance with feeding, diaper changes, and constant monitoring by caregivers. Human CNTNAP1 mutations are associated with hypomyelinating neuropathy-3, a condition leading to severe neurological dysfunction related to myelin sheath development.</span></p>
<p><span style="font-size: 15px;">In a recent study, Dr. Manzoor Bhat and his team at the University of Texas Health Science Center at San Antonio made a discovery that offers hope for these severely affected children through <a href="/gene-therapy" target="_blank" rel="noopener"><strong><span style="color: #0000ff;">gene therapy</span></strong></a>. The research findings were published online on October 19, 2023, in the journal Cell Reports under the title &#8220;Mouse models of human CNTNAP1-associated congenital hypomyelinating neuropathy and genetic restoration of murine neurological deficits.&#8221;</span></p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-162" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/11/1-s2.0-S221112472301286X-fx1_lrg.jpg" alt="" width="586" height="586" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/11/1-s2.0-S221112472301286X-fx1_lrg.jpg 996w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/11/1-s2.0-S221112472301286X-fx1_lrg-300x300.jpg 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/11/1-s2.0-S221112472301286X-fx1_lrg-150x150.jpg 150w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/11/1-s2.0-S221112472301286X-fx1_lrg-768x768.jpg 768w" sizes="(max-width: 586px) 100vw, 586px" /></p>
<p><span style="font-size: 15px;">Bhat stated, &#8220;We obtained genetic information from these families and created transgenic mice models that could replicate the human mutations and diseases. Our mice exhibited phenotypes or weaknesses similar to these children.&#8221; Bhat is a leading figure in the field of neuron-glial biology, and his laboratory discovered the mouse Cntnap1 gene in 2001.</span></p>
<p><span style="font-size: 15px;">Transgenic animals refer to animals in which foreign genes are introduced into the genome. The transgenic mice developed by Bhat&#8217;s lab carry both normal copies of the Cntnap1 gene and mutant copies reflecting the mutations observed in these children. Bhat explained, &#8220;We can control when the normal gene is turned on. It turns out we can use the normal gene to rescue the neural defects in these mice.&#8221;</span></p>
<p><span style="font-size: 15px;">In a series of experiments, the researchers activated the normal Cntnap1 gene in mice at birth, five days after birth, two weeks after birth, one month after birth, and three months after birth. The earlier the normal <a href="/gene-therapy/search?key=Cntnap1&amp;ty=product" target="_blank" rel="noopener"><strong><span style="color: #0000ff;">Cntnap1 gene</span></strong></a> was activated, the quicker the mice&#8217;s condition improved, and the rescue was more thorough.</span></p>
<p><span style="font-size: 15px;">Bhat noted, &#8220;The longer we waited, the worse the condition of these mice. This is because the Cntnap1 gene produces a protein that drives neural impulse conduction. If we wait for a month or two, neural function becomes weak, muscles become weak, and the mice can&#8217;t maintain motor coordination.&#8221;</span></p>
<p><span style="font-size: 15px;">The mice were placed on beams to measure their movement. Over time, they navigated the beam more easily after the activation of the normal Cntnap1 gene, as the protein produced by the normal gene improved neural signal conduction.</span></p>
<p><span style="font-size: 15px;">The next phase of this new research involves injecting a virus into Cntnap1 mutant mice that can produce the Cntnap1 protein. If preclinical studies show promising results, the next step will be gene therapy for children.</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Chang, Cheng, et al. &#8220;Mouse models of human CNTNAP1-associated congenital hypomyelinating neuropathy and genetic restoration of murine neurological deficits.&#8221; Cell Reports 42.10 (2023).</span></p>
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		<title>AAV Gene Therapy for Alcoholism: One-Time Treatment, Long-Term Effectiveness</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/aav-gene-therapy-for-alcoholism-one-time-treatment-long-term-effectiveness/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Thu, 05 Oct 2023 10:44:27 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[AAV]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<guid isPermaLink="false">https://www.creative-biolabs.com/blog/gene-therapy/?p=155</guid>

					<description><![CDATA[Researchers from Ohio State University, Oregon Health and Science University, Wake Forest School of Medicine, and the University of California, San Francisco, have published a research paper in the prestigious medical journal<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/aav-gene-therapy-for-alcoholism-one-time-treatment-long-term-effectiveness/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p><span style="font-size: 15px;">Researchers from Ohio State University, Oregon Health and Science University, Wake Forest School of Medicine, and the University of California, San Francisco, have published a research paper in the prestigious medical journal Nature Medicine titled &#8220;GDNF gene therapy for alcohol use disorder in male non-human primates.&#8221;</span></p>
<p><span style="font-size: 15px;">This study in non-human primates, specifically rhesus macaques, demonstrates that the delivery of human Glial-Derived Neurotrophic Factor (hGDNF) to the Ventral Tegmental Area (VTA) of the midbrain using adeno-associated virus serotype 2 (AAV2) can reduce alcohol consumption and prevent relapse of alcohol cravings after abstinence.</span></p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-157" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML.png" alt="" width="745" height="696" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML.png 1776w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML-300x280.png 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML-1024x956.png 1024w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML-768x717.png 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/10/41591_2023_2463_Fig1_HTML-1536x1434.png 1536w" sizes="(max-width: 745px) 100vw, 745px" /></p>
<p><span style="font-size: 15px;">Professor Krystof Bankiewicz, the corresponding author of the paper, stated that this <a href="/gene-therapy/" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene therapy</strong></span></a> targets changes in dopamine function within the midbrain&#8217;s edge reward pathway caused by chronic alcohol consumption. Experimental results show that this gene therapy can prevent the relapse of alcohol cravings after abstinence, potentially offering a one-time, sustained treatment for severe alcohol addiction, also known as Alcohol Use Disorder (AUD).</span></p>
<p><span style="font-size: 15px;">Excessive alcohol consumption alters the release of dopamine, a neurotransmitter, in the Ventral Tegmental Area (VTA) of the edge reward pathway in the midbrain. As AUD progresses, these changes become more pronounced and include reduced dopamine release, decreased dopamine receptor sensitivity, and increased dopamine uptake. These changes result in dopamine levels in the pathway being lower than normal. This &#8220;hypodopaminergic&#8221; state compels individuals to resume drinking after a period of abstinence. Currently, there are no treatments or medications targeting this pathway.</span></p>
<p><span style="font-size: 15px;">In this study, the research team conducted experiments on rhesus macaques, which were accustomed to consuming 4% alcohol before treatment. In the treatment group, AAV2-hGDNF was injected into the VTA of eight macaques, leading to the expression of Glial-Derived Neurotrophic Factor. Another four macaques received sterile saline injections using the same procedure as a control group.</span></p>
<p><span style="font-size: 15px;">Before treatment, these macaques were accustomed to drinking 4% alcohol. After treatment, the treatment group of macaques exhibited sustained expression of human Glial-Derived Neurotrophic Factor (hGDNF) in their brains. Over the subsequent 12 months, they showed no relapse in drinking behavior during repeated periods of abstinence and alcohol reintroduction challenges. This behavioral change was accompanied by neurophysiological regulation of ventral tegmental area (VTA) dopamine signaling, which counteracted the hypodopaminergic signaling state associated with chronic alcohol use. This suggests that <a href="/gene-therapy/search?key=GDNF&amp;ty=product" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>GDNF</strong></span></a> gene therapy targeting the VTA can reduce alcohol consumption and prevent relapse of alcohol cravings, potentially offering a promising treatment strategy for Alcohol Use Disorder (AUD).</span></p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Ford, Matthew M., et al. &#8220;GDNF gene therapy for alcohol use disorder in male non-human primates.&#8221; Nature medicine 29.8 (2023): 2030-2040.</span></p>
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		<title>New Gene Therapy Offers Hope for Treating Chronic Pain by Reducing Sodium Ion Levels</title>
		<link>https://www.creative-biolabs.com/blog/gene-therapy/new-gene-therapy-offers-hope-for-treating-chronic-pain-by-reducing-sodium-ion-levels/</link>
		
		<dc:creator><![CDATA[biolabs]]></dc:creator>
		<pubDate>Fri, 01 Sep 2023 05:42:50 +0000</pubDate>
				<category><![CDATA[Gene Therapy News]]></category>
		<category><![CDATA[Gene Therapy Research]]></category>
		<category><![CDATA[Chronic Pain]]></category>
		<category><![CDATA[Gene Therapy]]></category>
		<category><![CDATA[NaV1.7]]></category>
		<category><![CDATA[Sodium Ion Channels]]></category>
		<guid isPermaLink="false">http://www.creative-biolabs.com/blog/gene-therapy/?p=149</guid>

					<description><![CDATA[Innovative gene therapy may hold the key to treating chronic pain in humans by reducing sodium ion levels, a recent study published in the international journal &#8220;Proceedings of the National Academy of<a class="moretag" href="https://www.creative-biolabs.com/blog/gene-therapy/new-gene-therapy-offers-hope-for-treating-chronic-pain-by-reducing-sodium-ion-levels/">Read More...</a>]]></description>
										<content:encoded><![CDATA[<p>Innovative gene therapy may hold the key to treating chronic pain in humans by reducing sodium ion levels, a recent study published in the international journal &#8220;Proceedings of the National Academy of Sciences&#8221; reveals. Researchers from institutions such as New York University have developed a novel gene therapy approach that indirectly regulates specific sodium ion channels, potentially alleviating chronic pain in individuals.</p>
<p><img decoding="async" loading="lazy" class="aligncenter wp-image-150" src="http://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/09/keyimage.jpg" alt="" width="679" height="604" srcset="https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/09/keyimage.jpg 3856w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/09/keyimage-300x267.jpg 300w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/09/keyimage-768x683.jpg 768w, https://www.creative-biolabs.com/blog/gene-therapy/wp-content/uploads/sites/5/2023/09/keyimage-1024x911.jpg 1024w" sizes="(max-width: 679px) 100vw, 679px" /></p>
<p>Chronic pain is a significant public health issue, affecting approximately one-third of the US population. Scientists have been eager to develop more effective and safer analgesics than opioid drugs. Sodium ion channels play a crucial role in the generation and transmission of pain because of their vital role in communication between nerve cells or neurons. A specific sodium ion channel known as NaV1.7 has emerged as a potential target for treating human pain, as previous research has demonstrated its importance in patients with rare genetic pain disorders.</p>
<p>In some families, genetic mutations in the gene encoding NaV1.7 lead to an influx of sodium ions into cells, resulting in intense pain, while in other families, mutations that block NaV1.7 lead to complete painlessness. For years, scientists have been researching and attempting to develop new pain therapies that selectively block NaV1.7, but with limited success. Researchers like Khanna and colleagues took a different approach, not by blocking NaV1.7 directly but by modulating it through a protein called <span style="color: #0000ff;"><a style="color: #0000ff;" href="/gene-therapy/googlesearch?key=CRMP2&amp;ty=google" target="_blank" rel="noopener"><strong>CRMP2</strong></a></span>.</p>
<p>According to Khanna, CRMP2 can interact with sodium ion channels and regulate their activity, allowing more or fewer sodium ions to enter the channels. By inhibiting the interaction between Nav1.7 and CRMP2, it may be possible to reduce sodium ion influx, calming down neurons and thereby reducing pain sensations in the body. Previously, researchers developed a small molecule that indirectly regulated Nav1.7 expression by targeting CRMP2. This compound successfully controlled pain in cell and animal models, and current research is exploring its application in humans.</p>
<p>While this compound has shown promise, a critical question remained: why does CRMP2 interact exclusively with NaV1.7 sodium ion channels and not with the other eight sodium ion channels in the same family? In this study, researchers identified a specific region in NaV1.7 where CRMP2 protein binds to regulate its activity. This region appears unique to NaV1.7, as CRMP2 does not directly bind to other sodium ion channels. This discovery raised excitement among researchers because removing this specific portion of the NaV1.7 channel would result in the loss of CRMP2&#8217;s regulatory effect.</p>
<p>To restrict the interaction between CRMP2 and NaV1.7, researchers created a peptide from the channel that corresponded to the region where CRMP2 binds. They then inserted this peptide into adenoviruses, which were used to transport it into neurons and inhibit the function of NaV1.7. The use of viruses to deliver genetic material into cells is a primary method in <a href="/gene-therapy/" target="_blank" rel="noopener"><span style="color: #0000ff;"><strong>gene therapy</strong></span></a>, which has already been successful in treating blood disorders, eye conditions, and other rare diseases.</p>
<p>Researchers injected these engineered viruses into mice experiencing various forms of pain, including touch, heat, cold, and chemotherapy-induced peripheral neuropathy. After one to ten days, the researchers evaluated the animals and found that their pain sensations had reversed. Khanna stated that they have discovered a new approach that uses engineered viruses containing a small piece of genetic material that everyone possesses to infect neurons effectively, providing an effective treatment for pain. This represents a significant advancement in the field of gene therapy for chronic pain and is just one of the latest examples of its potential applications.</p>
<p>Researchers replicated their findings of NaV1.7 inhibition in multiple species, including rodents, primates, and human cells. While further research is needed, this study holds promise, indicating that their approach could potentially translate into therapeutic methods for humans. There is an urgent need to develop new pain therapies, especially for cancer patients suffering from chemotherapy-induced neuropathy. The long-term goal of researchers is to develop a novel gene therapy that can better treat these painful conditions and improve patients&#8217; quality of life. In conclusion, the study&#8217;s results support the idea that the CRMP2 regulatory sequence (CRS) domain may be a targetable region for treating chronic neuropathic pain in humans.</p>
<p><span style="color: #808080;">Reference</span></p>
<p><span style="color: #808080; font-size: 14px;">1. Gomez, Kimberly, et al. &#8220;Identification and targeting of a unique NaV1. 7 domain driving chronic pain.&#8221; Proceedings of the National Academy of Sciences 120.32 (2023): e2217800120.</span></p>
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