Key result
CRISPR technologies drive emerging therapies for hypercholesterolemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.
Why the study?
CRISPR technologies have progressed rapidly over the past decade, transforming biomedical research and yielding new therapies poised to enter the clinic for cardiovascular diseases.
CRISPR technologies are advancing rapidly, offering transformative potential for cardiovascular basic science research and emerging clinical therapies for conditions like hypercholesterolaemia and transthyretin amyloidosis.
CRISPR applications in cardiology remain investigational; extends research avenues but leaves open clinical translation and safety data.
CRISPR technologies have progressed by leaps and bounds over the past decade, not only having a transformative effect on biomedical research but also yielding new therapies that are poised to enter the clinic. In this review, I give an overview of (i) the various CRISPR DNA-editing technologies, including standard nuclease gene editing, base editing, prime editing, and epigenome editing, (ii) their impact on cardiovascular basic science research, including animal models, human pluripotent stem cell models, and functional screens, and (iii) emerging therapeutic applications for patients with cardiovascular diseases, focusing on the examples of hypercholesterolaemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.
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Kiran Musunuru (2022) conducted a review in Cardiovascular diseases. CRISPR technologies was evaluated. CRISPR DNA-editing technologies are transforming cardiovascular research and yielding emerging therapies for hypercholesterolaemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.
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