The first vector-delivered gene therapies to receive FDA approval for clinical use were Luxturna,1 Spark Therapeutics’ vision loss cure, and Zolgensma,2 Novartis’ spinal muscular atrophy treatment.
Osaka, Japan - A research team at The University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...
PHILADELPHIA--(BUSINESS WIRE)--VintaBio, a biotech manufacturer specializing in high-yield, high-purity viral vectors for gene therapy, today presented data further demonstrating the advantages and ...
Adeno-associated virus (AAV) vectors have become widely recognized as a fundamental tool in gene therapy, primarily due to their potential as a delivery system for the treatment of genetic disorders.
What Are AAV Vector-Based Therapies? Gene therapies are groundbreaking approaches utilizing viral and non-viral vectors. These vectors introduce therapeutic genes into patient cells to treat diseases, ...
In the mid-1990s, Hiroyuki Nakai, now an adeno-associated virus (AAV) researcher at the Oregon Health and Science University, moved to the United States. Nakai quickly became fascinated with the ...
DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...
Gene therapy can target the underlying causes of disease and even cure previously untreatable conditions. But despite this potential, the market remains small with only a handful of approved products.
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