Targeting SR Proteins Improves SMN Expression in Spinal Muscular Atrophy Cells
Spinal muscular atrophy (SMA) is one of the most common inherited causes of pediatric mortality. SMA is caused by deletions or mutations in the survival of motor neuron 1 (SMN1) gene, which results in SMN protein deficiency. Humans have a centromeric copy of the survival of motor neuron gene, SMN2,...
| Main Authors: | , , , |
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| Format: | Online |
| Language: | English |
| Published: |
Public Library of Science
2014
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4266657/ |