Promoting expression in gene therapy: more is not always better
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by loss-of-function of SMN1. SMA is characterized by degeneration of motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. One of three currently available treatments is onasemnogene...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Springer Nature
2024-02-01
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| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.1038/s44321-024-00036-y |