Article

Efficient and durable gene activation by Cas9-mediated epigenome editing in vivo

2024-05-05

Abstract excerpt

Epigenome editing technology holds great promise for treating diverse genetic disorders. While a series of advances has been made on epigenetic silencing using programmable editors, little progress has been made in leveraging epigenetic activation for therapeutic application. Here we demonstrate epigenetic activation of the LAMA1 gene for the treatment of LAMA2-CMD, a severe congenital muscle dystrophy (CMD) cause...

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Literature Corpus work: 5489f0d3-9030-54a5-be87-75a59c3cf466
DOI: 10.1101/2024.05.03.592438

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