AAV Vector Design for Gene Therapy
Compared with other gene delivery vectors, recent advancements in various adeno-associated virus (AAV) engineering have greatly expanded their applications for gene therapy as an ideal gene vehicle. Creative Biolabs has successfully established a state-of-art gene-therapy platform to offer diverse services form vector design to efficacy testing, to satisfy multiple research and clinical application demands of worldwide clients.
AAV Vector-Based Gene Therapy
Gene therapy is a method of human disease treatment by introducing or changing a therapeutic genetic fragment in host cells, which has revolutionized the current treatment state of some incurable diseases. AAV is a kind of non-enveloped virus with a linear single-stranded DNA genome. The properties including non-pathogenicity, low-immunogenicity, non-dividing and dividing cells infectivity, etc., make AAVs attractive and promising vehicles for gene transfer and therapy. AAV vector-based gene delivery has overcome the limitations of the conventional drug for "undruggable" targets by gene addition, replacement, editing and silencing. Currently, three AAV vector-based gene therapy products have been approved by the Food and Drug Administration (FDA), and more than 100 AAV vectors related clinical trials are under evaluation, aiming to offer "one-and-done" therapeutic solutions for patients who suffer from some refractory diseases like hemophilia, neurological disorders, arthritis, etc.
Figure 1. Adeno-associated virus (AAV): a promising vector for gene therapy.
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The potential application of AAV vectors has been expanding with the development of engineering and modification strategies. A list of human genetic disorders is extensively and intensively researched and evaluated by AAV vector-based gene therapies including central nervous system (CNS) diseases, ocular diseases, liver-directed diseases, and other hereditary diseases. Creative Biolabs provides professional AAV vector design services to benefit clinical gene therapies for a vast range of human disorders, as follows:
- CNS Diseases
- Liver Directed Diseases
- Ocular Diseases
- Other Hereditary Diseases
CNS disease or neurological disorder still remains to be one of the most challenging diseases; the main reason is that the blood-brain barrier hinders the passage and delivery of many important diagnostic and therapeutic molecules to the brain. Recent gene therapies provide a new promising approach for the treatment of CNS diseases by blocking pathogenic proteins or promoting the missing protein expression. The exhibiting properties of AAV together with advanced vector design and administration strategies enable AAV vectors to be an excellent tool for gene delivery to the CNS. AAV vectors are commonly used delivery systems for gene therapy of diverse CNS disorders.
Livers play a significant role in regulating metabolic activities and signal-transducing pathways. And the abnormality of hepatocytes is associated with a wide range of inherited genetic/metabolic disorders, suggesting liver/hepatocyte is an important target for gene therapy of human hereditary diseases. Preclinical and clinical successes by AAV vector-based gene therapies in hemophilia B further stimulate the development of liver-directed AAV gene therapy. There are a number of clinical trials that are under-evaluated for the treatment of a series of liver disorders.
The first gene therapy approved by FDA is Luxturna (voretigene neparvovec-rzyl), an AAV vector-based gene therapy for the treatment of patients with retinal dystrophy or hereditary blindness. The identification of pathogenicity genes, retina tropism of AAVs and advanced administration strategies spur AAV-based ophthalmic gene therapy to be more efficient and safer, opening doors for the development of further ophthalmic gene therapy.
The applications of AAV-based gene therapy are not only limited to the therapies for the above diseases, but also for the treatment of some other hereditary diseases. By introducing new genetic instructions into specific tissues to compensate for missing genes or replacing/silencing abnormal genes, AAV vector-based gene delivery has been extensively utilized for hereditary diseases therapies and has shown favorably therapeutic efficacy in a range of animal models.
As a biotech company dedicated to human health improvement, Creative Biolabs provides one-stop customized AAV vector design services for a list of disorders, to fulfill the demands of basic research as well as therapeutic applications. You can find a target disease in details, or contact us for your customized experimental design.
