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Duchenne muscular dystrophy awaits gene therapy

Nature Biotechnology volume 37, pages 335–337 (2019)Cite this article

Solid’s gene therapy disappoints in the clinic, but other gene therapies and exon-skipping agents continue their march, with CRISPR on the way.

The prospect of finding disease-modifying drugs to supplement or replace the defective dystrophin protein in DMD patients had seemed within reach in recent years, as companies pursued moving gene therapies and exon-skipping agents into the clinic. But so far their success in gaining sufficient product expression in muscle has been limited and, in some cases, controversial.

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  1. Brighton, UK

    Dan Jones

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Jones, D. Duchenne muscular dystrophy awaits gene therapy. Nat Biotechnol 37, 335–337 (2019). https://doi.org/10.1038/s41587-019-0103-5

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