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Developing therapies for spinal muscular atrophy
Spinal muscular atrophy is an autosomal-recessive pediatric neurodegenerative disease characterized by loss of spinal motor neurons. It is caused by mutation in the survival of motor neuron 1 gene (SMN1) leading to loss of function of the full-length SMN protein. SMN has a number of functions in neu...
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| Vydáno v: | Ann N Y Acad Sci |
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| Hlavní autoři: | , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
2015
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4713374/ https://ncbi.nlm.nih.gov/pubmed/26173388 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/nyas.12813 |
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