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.

ग्रंथसूची विवरण
मुख्य लेखकों: Davies, KE, Chamberlain, JS
स्वरूप: Journal article
भाषा:English
प्रकाशित: Springer Nature 2019
विवरण
सारांश: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.