Splicing therapy for neuromuscular disease.
Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two of the most common inherited neuromuscular diseases in humans. Both conditions are fatal and no clinically available treatments are able to significantly alter disease course in either case. However, by manipulation of pre-m...
| Main Authors: | , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2013
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