AAV Gene Therapy Prevents and Reverses Heart Failure in A Murine Knockout Model of Barth Syndrome.

Conclusions: TAZ-KO and TAZ-CKO mice recapitulate many of the key clinical features of BTHS. AAV-mediated gene replacement is efficacious when a sufficient fraction of CMs are transduced.
PMID: 32146862 [PubMed - as supplied by publisher]

Source: Circulation Research - March 8, 2020 Category: Cardiology Authors: Wang S, Li Y, Xu Y, Ma Q, Lin Z, Schlame M, Bezzerides VJ, Strathdee D, Pu WT Tags: Circ Res Source Type: research