Subsequently, it increases the risk of heart failure, stroke, and sudden cardiac arrest.

Though rare, HCM, a primary myocardial genetic disorder, causes left ventricular hypertrophy (LVH) that increases myocardial fibrosis. Image Credit: Catalin Rusnac / Shutterstock Background Study: Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice. Note that clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) uses CRISPR sequences, a family of deoxyribonucleic acid (DNA) sequences found in the genomes of some bacteria, as a guide to identify and cleave other complementary DNA sequences.

Ii) a potent Cas9 nuclease delivered by an adeno-associated virus (AAV) vector.

They used a previously constructed murine model of HCM, designated as R403Q-129SvEv, to evaluate two different genetic therapies, as follows:

In a recent study published in the journal Nature Medicine, researchers pursued one-time cures for hypertrophic cardiomyopathy (HCM).

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