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.

Библиографические подробности
Главные авторы: Davies, KE, Chamberlain, JS
Формат: Journal article
Язык:English
Опубликовано: Springer Nature 2019
Описание
Итог: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.