Enhanced Cytosine Base Editing for Duchenne Muscular Dystrophy - CRISPR Medicine
Chinese researchers developed aTdCBE, a novel cytosine base editor lacking adenosine deaminase activity, to correct exon 55 splicing in a DMD mouse model. In vivo delivery resulted in over 40% DNA base editing at the exon 55 splice acceptor site, nearly 99% dystrophin-positive muscle fibres, and 60% dystrophin expression restoration, demonstrating efficient gene editing and significant dystrophin restoration.
Reference News
Chinese researchers developed aTdCBE, a novel cytosine base editor lacking adenosine deaminase activity, to correct exon 55 splicing in a DMD mouse model. In vivo delivery resulted in over 40% DNA base editing at the exon 55 splice acceptor site, nearly 99% dystrophin-positive muscle fibres, and 60% dystrophin expression restoration, demonstrating efficient gene editing and significant dystrophin restoration.