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Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)
Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease. It is the first genetic cause of infant mortality. It is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the reduction of SMN protein. The most striking component is the loss of alpha motor neuro...
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| Main Authors: | , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
John Wiley & Sons Ltd
2014
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3930406/ https://ncbi.nlm.nih.gov/pubmed/24400925 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jcmm.12224 |
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