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

Wang S, Li Y, Xu Y, Ma Q, Lin Z, Schlame M, Bezzerides VJ, Strathdee D, Pu WT
Circ Res. 2020 Mar 9. doi: 10.1161/CIRCRESAHA.119.315956. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID	Plasmid	Purpose
139979	pCAG/hTAZ-FL	Expresses full length human Tafazzin with codon modified for cloning. Transgene flanked by AAV2 ITR
139980	pCAG/GFP	expresses GFP under the promoter of CAG. Transgene flanked by AAV2 ITR
139981	pCAG/Luci	Expresses luciferase under the promoter of CAG. Transgene flanked by AAV2 ITR