Immune Responses to AAV in Clinical Trials

Abstract excerpt

Findings in the first clinical trial in which an adeno-associated virus (AAV) vector was introduced into the liver of human subjects highlighted an issue not previously identified in animal studies. Upon AAV gene transfer to liver, two subjects developed transient elevation of liver enzymes, likely as a consequence of immune rejection of transduced hepatocytes mediated by AAV capsid-specific CD8(+) T cells....

Topics

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

• Share to Hemophilia Treatment and Research
• Share to Viral Infectious Diseases and Gene Expression in Insects
• Share to Virus-based gene therapy research