Better Outfitted and Screened Viral Vectors Are Safer and More Effective
Better Outfitted and Screened Viral Vectors Are Safer and More Effective
Adeno-associated virus (AAV) vectors are the leading vectors for gene delivery. Two AAV-based gene therapies have been approved by the U.S. Food and Drug administration, and dozens more are in clinical trials. However, AAV vectors pose challenges including costly manufacture, high percentages of empty capsids, and safety issues associated with high-dose regimens. To address this last challenge, developers are working to improve the impact of each AAV particle so that fewer particles will be required. [Dr. Microbe/Getty Images]