It arises from the dominant missense allele that alters the contractile protein cardiac myosin heavy chain ( Myhc) in humans.Īll the factors that influence HCM emergence remain unknown however, it is known that pathogenic myosin allelic variants produce symptomatic HCM early in adulthood when LVH and symptoms are absent, presenting a window of opportunity for potential interventions to limit or prevent disease.Īlthough medications and devices could help reduce HCM symptoms, they cannot prevent heart failure, the leading cause of premature death in HCM patients. 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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