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.
| Auteurs principaux: | , |
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| Format: | Journal article |
| Langue: | English |
| Publié: |
Springer Nature
2019
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