CRISPR BASED GENE EDITING FOR INHERITED CARDIOMYOPATHIES: A SCIENTIFIC MEDICAL ANALYSIS AND TRANSLATIONAL FRAMEWORK

Abstract

Dar, S. (2026). CRISPR-Based Gene Editing for Inherited Cardiomyopathies: A Scientific Medical Analysis and Translational Framework. DOI: https://doi.org/10.5281/zenodo.22132605. Affiliations: Director, Medical Wing, MKET Canada; Medical Legal Editor, CLRJ Toronto. Summary In this comprehensive scientific analysis, Dr. Shehneela Dar (2026) explores the emerging role of CRISPR‑based gene editing as a therapeutic strategy for inherited cardiomyopathies, including hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), and arrhythmogenic cardiomyopathy (ACM). The paper synthesizes current genomic knowledge and highlights how CRISPR‑Cas9, adenine base editors (ABE), and RNA‑targeting CRISPR‑Cas13 systems can correct or suppress pathogenic variants responsible for cardiomyopathic remodelling. Dr. Shehneela Dar (2026) reviews recent experimental evidence demonstrating successful in vivo correction of MYH7 mutations, allele‑specific RNA cleavage of mutant transcripts, and prevention of disease progression in animal models. The paper also examines delivery platforms such as AAV9 vectors, evaluates safety concerns including off‑target effects and immunogenicity, and discusses translational barriers to clinical implementation. The study concludes with detailed recommendations for research, regulatory policy, and ethical governance to support safe and equitable adoption of genomic therapies in cardiovascular medicine. Evaluation Dr. Shehneela Dar’s (2026) work is notable for its depth, clarity, and interdisciplinary integration. The paper demonstrates strong scientific rigour by combining molecular genetics, cardiology, and translational medicine. It provides a balanced evaluation of CRISPR technologies, acknowledging both their therapeutic promise and their limitations. The discussion of allele‑specific editing, RNA‑targeting CRISPR systems, and multi‑vector delivery platforms reflects cutting‑edge developments in genomic therapy. The inclusion of ethical and regulatory considerations strengthens the paper’s relevance for policymakers and clinical researchers. Overall, this work represents a significant scholarly contribution to the emerging field of cardiovascular gene editing. Relevance to Research Topic This source is foundational for any research on CRISPR‑based therapies for inherited cardiomyopathies. It provides essential insights into the molecular mechanisms of cardiomyopathy, the therapeutic potential of gene editing, and the translational challenges associated with clinical implementation. The paper’s emphasis on MYH7 correction, allele‑specific RNA editing, and AAV9‑mediated delivery makes it highly relevant for researchers developing next‑generation genomic therapies. Its policy and ethical recommendations are directly applicable to precision cardiology initiatives in Canada and worldwide. Keywords: CRISPR gene editing; inherited cardiomyopathies; hypertrophic cardiomyopathy (HCM); dilated cardiomyopathy (DCM); arrhythmogenic cardiomyopathy (ACM); MYH7 mutation; CRISPR‑Cas9; adenine base editing (ABE); CRISPR‑Cas13d; allele‑specific RNA editing; AAV9 delivery; genomic therapy; precision cardiology; translational genomics; cardiac molecular medicine.