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BioWorld - Friday, April 10, 2026
Breaking News: Regeneration in mammals is controlled by environmental conditionsSee today's BioWorld Science
Home » CRISPR-mediated utrophin upregulation preclinically improves DMD
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Neurology/psychiatric

CRISPR-mediated utrophin upregulation preclinically improves DMD

April 1, 2026
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Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect.
BioWorld Science Genetic/congenital Neurology/psychiatric

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