Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing
Duchenne muscular dystrophy (DMD) is a currently incurable X-linked neuromuscular disorder, characterized by progressive muscle wasting and premature death, typically as a consequence of cardiac failure. DMD-causing mutations in the dystrophin gene are highly diverse, meaning that the development of...
| Main Authors: | , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
Taylor and Francis
2021
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