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A Cell System for Phenotypic Screening of Modifiers of SMN2 Gene Expression and Function
Spinal muscular atrophy (SMA) is an inherited neurodegenerative disease caused by homozygous inactivation of the SMN1 gene and reduced levels of the survival motor neuron (SMN) protein. Since higher copy numbers of the nearly identical SMN2 gene reduce disease severity, to date most efforts to devel...
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| Hauptverfasser: | , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Public Library of Science
2013
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3744461/ https://ncbi.nlm.nih.gov/pubmed/23967270 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0071965 |
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