GeneticsNaturePeer-reviewed
A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy in vivo.
Dwi U. Kemaladewi, Prabhpreet S. Bassi, Steven Erwood…·Ronald D. Cohn
This paper demonstrates that upregulating a disease modifier gene using CRISPR/dCas9 can rescue muscular dystrophy in mice, showing that muscle fibrosis may be reversible.
165 citationstop 2%