CRISPR and cardiovascular diseases
Kiran Musunuru
Abstract
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.
Topics & Concepts
CRISPRGenome editingInduced pluripotent stem cellTranslational researchZinc finger nucleaseComputational biologyDuchenne muscular dystrophyEpigenomeBioinformaticsBiologyMedicineGeneticsGeneEmbryonic stem cellBiotechnologyDNA methylationGene expressionCRISPR and Genetic EngineeringGenetics, Aging, and Longevity in Model OrganismsViral Infections and Immunology Research