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Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy
Spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality. SMA is caused by loss of functional survival motor neuron 1 (SMN1), resulting in death of spinal motor neurons. Current therapeutic research focuses on modulating the expression of a partially functioning copy gene,...
Gorde:
| Egile Nagusiak: | , , , |
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| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
Oxford University Press
2009
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2649020/ https://ncbi.nlm.nih.gov/pubmed/19074460 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddn426 |
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