A Comprehensive Review on CRISPR Technology in the Treatment and Understanding of Cardiacmyopathy
Gottipati Jayanth *
Govt. Siddhartha Medical College, Dr. N.T.R University of Health Sciences, Vijayawada, Andhra Pradesh, India.
*Author to whom correspondence should be addressed.
Abstract
Cardiac myopathy, encompassing hypertrophic, dilated, restrictive, and arrhythmogenic forms, represents a significant challenge due to its complex genetic underpinnings and limited curative treatments.
Advances in CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology offer transformative potential in understanding and treating these disorders. CRISPR-Cas9 enables precise gene editing, addressing pathogenic mutations such as MYH7 in hypertrophic cardiomyopathy and TTN in dilated cardiomyopathy.
Emerging techniques like base and prime editing enhance accuracy while minimizing off-target effects. Preclinical models and in vivo studies have demonstrated the utility of CRISPR in creating disease models, correcting genetic defects, and exploring therapeutic interventions. However, challenges including delivery mechanisms, ethical considerations, and long-term safety must be addressed.
This review explores the therapeutic promise and limitations of CRISPR in cardiac myopathy, highlighting its role in precision medicine and its potential to revolutionize cardiovascular treatment.
Keywords: Cardiac myopathy, CRISPR-Cas9, gene editing, precision medicine, genetic mutations