Healing a Heart Through Genetic Intervention.

Abstract

A recent manuscript from Eric Olson’s group outlines the development of an important new tool that should catalyze the cardiovascular community’s ability to create new animal models containing genetically engineered genes and proteins specifically in the cardiomyocyte (CM) population. Olson and colleagues utilized a CM-specific promoter to express a critical component of the <i><u>C</u></i>lustered <i><u>R</u></i>egularly <i><u>I</u></i>nterspaced <i><u>S</u></i>hort <i><u>P</u></i>alindromic <i><u>R</u></i>epeats (CRISPR)-associated (Cas)9 genomic editing system. The α myosin heavy chain gene (<i>myh6</i>) promoter was used to drive high levels of CM-specific Cas9 expression. Subsequently, Adeno-Associated Virus (AAV) was used to deliver single-guide RNA (sgRNA) against the <i>myh6 locus</i> and they were able to show efficient gene editing at the locus, establishing proof of principle for what the authors term “cardioediting,” a strategy for revising a specific locus in the CMs at any time post birth.