Therapeutic expression of human clotting factors IX and × following adeno‐associated viral vector‐mediated intrauterine gene transfer in early‐gestation fetal macaques

Abstract excerpt

ABSTRACT Adeno‐associated viral vectors (AAVs) achieve stable therapeutic expression without long‐term toxicity in adults with hemophilia. To avert irreversible complications in congenital disorders producing early pathogenesis, safety and efficacy of AAV‐intrauterine gene transfer (IUGT) requires assessment We therefore performed IUGT of AAV5 or ‐8 with liver‐specific promoter‐1 encoding either human coagulation...

Topics

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

• Share to CRISPR and Genetic Engineering
• Share to Parvovirus B19 Infection Studies
• Share to Virus-based gene therapy research