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.
Автори: | , |
---|---|
Формат: | Journal article |
Мова: | English |
Опубліковано: |
Springer Nature
2019
|