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Cas13b-mediated RNA targeted therapy alleviates genetic dilated cardiomyopathy in mice

4 Jan 2024

Abstract excerpt

Abstract Background Recent advances in gene editing technology have opened up new avenues for in vivo gene therapy, which holds great promise as a potential treatment method for dilated cardiomyopathy (DCM). The CRISPR-Cas13 system has been shown to be an effective tool for knocking down RNA expression in mammalian cells. PspCas13b, a type VI-B effector that can be packed into adeno-associated viruses and improve...

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PMID: 38178244
DOI: 10.1186/s13578-023-01143-y

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