Modulating role of RNA structure in alternative splicing of a critical exon in the spinal muscular atrophy genes
Humans have two nearly identical copies of the survival motor neuron (SMN ) gene, SMN1 and SMN2. Homozygous loss of SMN1 causes spinal muscular atrophy (SMA). SMN2 is unable to prevent the disease due to skipping of exon 7. Using a systematic approach of in vivo selection, we have previously demonst...
Main Authors: | , , |
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Format: | Online |
Language: | English |
Published: |
Oxford University Press
2007
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1802598/ |