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An engineered truncated gene derived from the dystrophin-related protein (utrophin), prevents pathology without an immune response in an animal model of Duchenne muscular dystrophy (DMD) gene therapy.

Original publication

DOI

10.1038/s41591-019-0604-2

Type

Journal article

Journal

Nature Medicine

Publisher

Springer Nature

Publication Date

07/10/2019

Volume

25

Pages

1473 - 1474

Keywords

FFR