Surrogate gene therapy for muscular dystrophy

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.

Sonraí bibleagrafaíochta
Príomhchruthaitheoirí: Davies, KE, Chamberlain, JS
Formáid: Journal article
Teanga:English
Foilsithe / Cruthaithe: Springer Nature 2019