The team led by Bejamine Deverman at the Broad Institute of MIT and Harvard published a research paper in the journal Science titled “An AAV capsid reprogrammed to bind human transferrin receptorRead More…
Breakthrough in ALS Treatment: Tofersen Significantly Slows Disease Progression in SOD1 Mutation Patient
Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig’s disease, is recognized by the World Health Organization (WHO) as one of the five major incurable diseases. It is the most common motorRead More…
Advancements in Gene Therapy: Ring Therapeutics Develops Anellovirus-Based Anellovector for Safe and Effective Treatment
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—usingRead More…
Revolutionizing Gene Therapy: Human Liver Model Advances Preclinical Testing
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,Read More…
PRINT: Enhancing Gene Therapy with Precision Retrotransposon Insertion
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 entireRead More…
New Gene Therapy Offers Hope for Treating Devastating Childhood Epilepsy: A Study from UCL Researchers
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.Read More…
Gene Therapy for Arrhythmogenic Cardiomyopathy: Pioneering Research from Utrecht University Medical Center
Recently, researchers from the Utrecht University Medical Center published a research paper in the Nature subsidiary journal, Nature Cardiovascular Research, titled: “Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.” TheRead More…
Gene Therapy Holds Promise for Paralysis Caused by CNTNAP1 Mutations
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. TheseRead More…
AAV Gene Therapy for Alcoholism: One-Time Treatment, Long-Term Effectiveness
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 journalRead More…
New Gene Therapy Offers Hope for Treating Chronic Pain by Reducing Sodium Ion Levels
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 “Proceedings of the National Academy ofRead More…