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Article

Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy

26 Jun 2019

Abstract excerpt

Many potentially therapeutic molecules have been identified for treating Duchenne muscular dystrophy. However, targeting those molecules only to sites of active pathology is an obstacle to their clinical use. Because dystrophic muscles become extensively inflamed, we tested whether expressing a therapeutic transgene in leukocyte progenitors that invade muscle would provide selective, timely delivery to diseased...

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PMID: 31243277
DOI: 10.1038/s41467-019-10614-1

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