Researchers at the Regenxbio pharmaceutical laboratory and American academics review the hepatic toxicity of adeno-associated viruses (AAV) in the context of human gene therapy, and ways to counteract it:
- systemically injected AAVs have a strong tropism for the liver, resulting in a high risk of complications given the very high doses used in current clinical protocols,
- the hu.32 and AAV5 capsids studied and modified by the researchers make it possible to detarget the liver while maintaining good clinical efficacy,
- this is even more true with the addition of NVG7 and NVG13 peptides.
These new vectors, which will need to prove themselves in clinical trials, offer hope in a field where safety issues are farfrom resolved.
