Hepatic Gene Transfer in Neonatal Mice by Adeno-Associated Virus Serotype 8 Vector

Abstract excerpt

For genetic diseases that manifest at a young age with irreversible consequences, early treatment is critical and essential. Neonatal gene therapy has the advantages of achieving therapeutic effects before disease manifestation, a low vector requirement and high vector-to-cell ratio, and a relatively immature immune system. Therapeutic effects or long-term rescue of neonatal lethality have been demonstrated in...

Topics

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

• Share to CRISPR and Genetic Engineering
• Share to Viral gastroenteritis research and epidemiology
• Share to Virus-based gene therapy research