NewsMOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPYThursday, April 9, 2026 · April 9, 2026
CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.
WHY IT MATTERS
Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of dystrophin. Upregulating utrophin, a dystrophin paralog, is a promising gene therapy approach. Here, we present a CRISPR-Cas9-based strategy to enhance utrophin expression by disrupting repressor binding sites. Us...
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