Comparison of Cardiac Specific Promoters to Liver-Specific miRNA Targets to Maximize Cardiac vs Liver Expression Following Intravenous AAVrh.10-Mediated Cardiac Gene Therapy

1518 Comparison of Cardiac Specific Promoters to Liver-Specific miRNA Targets to Maximize Cardiac vs Liver Expression Following Intravenous AAVrh.10-Mediated Cardiac Gene Therapy; Abhishek Bose, Neil R. Hackett, Nadir Khan-Yusufzai, Katie M. Stiles, Ronald G. Crystal. May 01, 2023 Volume 31Issue 4Supplement 1S1-794 26th Annual Meeting of the American Society of Gene & Cell Therapy. 

 Intravenous (IV) administration of a cardiotropic AAV vector is the simplest and most effective strategy to deliver a gene to the myocardium for the treatment of hereditary cardiac disease. The challenge in using the IV route is that a substantial proportion of AAV vectors distribute to the liver, resulting in high liver expression with risks of liver toxicity.

Comparison of Cardiac Specific Promoters to Liver-Specific miRNA Targets to Maximize Cardiac vs Liver Expression Following Intravenous AAVrh.10-Mediated Cardiac Gene Therapy