Efficient In Vivo Homology-Directed Repair Within Cardiomyocytes
Yanjiang Zheng, Nathan J. VanDusen, Catalina E. Butler, Qing Ma, Justin S. King, William T. Pu
Abstract
RISPR/Cas9-based genome editing technologies provide powerful tools for genetic manipulation. Cas9 in vivo genome editing of cardiomyocytes through nonhomologous end joining efficiently creates insertion-deletion mutations at guide RNA-targeted sites. 1 However, precise addition of new genetic information, or mutation correction, requires alternate strategies. Delivery of Cas9 and a homology directed repair (HDR) template using adeno-associated virus (AAV) was recently shown to enable creation of precise genomic edits, even within postmitotic cells. Animal experiments were approved by the Institutional Animal Care and Use Committee and adhered to institutional guidelines. The authors will provide data, results, and reagents on reasonable request.