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Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
The motor neuron disease spinal muscular atrophy (SMA) is caused by recessive, loss-of-function mutations of the survival motor neuron 1 gene (SMN1). Alone, such mutations are embryonically lethal, but SMA patients retain a paralog gene, SMN2, that undergoes alternative pre-mRNA splicing, producing...
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| Publicado no: | J Clin Invest |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Clinical Investigation
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6063504/ https://ncbi.nlm.nih.gov/pubmed/29985170 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI121658 |
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