Article

CRISPR/Cas13-mediated Dynamin 2 reduction therapy in a canine model of <i>DNM2</i> -related centronuclear myopathy

2025-09-05

Abstract excerpt

We determined the potential of CRISPR/Cas13 technology as a therapeutic approach for centronuclear myopathies (CNMs) by reducing the expression of a single protein, DNM2. CNMs are severe congenital rare muscle disorders that result in muscle hypotrophy and weakness, with no cure. CNMs frequently result from mutations in either BIN1 , MTM1 , or DNM2 genes, with DNM2 being a key GTPase that plays a pivotal role i...

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Literature Corpus work: cb11f029-95ae-58ab-adbf-d993a0236b89
DOI: 10.1101/2025.09.05.672814

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