Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors

Abstract excerpt

Over the last two decades, gene therapy vectors based on wild-type Adeno-associated viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into three approved gene therapy products. Concomitantly, a large body of preclinical work has illustrated the power and potential of engineered synthetic AAV capsids that often excel in terms of an organ or cell specificity, the efficiency of in...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to RNA Interference and Gene Delivery
• Share to Viral Infections and Immunology Research
• Share to Virus-based gene therapy research