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Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests


Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. 

The study, “The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy,” appeared in the journal Human Molecular Genetics.

Current dystrophin-replacement treatment approaches in DMD have limitations, such as not being applicable to specific subsets of patients, and gene therapies are troubled by low efficiency and poor targeting in fibrotic (scarred) muscle. They are not able to provide the same benefit of full-length dystrophin, the protein missing in these patients.

Raising Both Dystrophin And Utrophin Levels May Rescue Muscles In DMD, Mouse Study Suggests