Exploring Gene Editing for the Treatment of Heart Diseases

Abstract

Heart diseases can often be caused by genetic mutations—small errors in the DNA that affect how the heart functions. Scientists have created a powerful tool called CRISPR-Cas9, which works like tiny, precise scissors to cut DNA at specific spots. This allows them to fix these genetic mistakes by correcting faulty genes, adding missing parts, or changing single DNA bases that cause heart problems. For example, CRISPR-Cas9 can help treat hypertrophic cardiomyopathy, a condition where the heart muscle becomes too thick and doesn’t pump blood well. The system uses a guide RNA to lead the Cas9 protein to the exact place in the DNA that needs to be edited. Although this technology shows great promise and has been successfully tested in animals, there are still challenges. Scientists must ensure the treatment is safe and develop ways to deliver it effectively to heart cells. With ongoing research and improvements, CRISPR gene editing could change how genetic heart diseases are treated, offering new hope to many patients in the future.