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.

מידע ביבליוגרפי
Main Authors: Davies, KE, Chamberlain, JS
פורמט: Journal article
שפה:English
יצא לאור: Springer Nature 2019