Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
Author:
Funder
SMA Foundation
The SMA Research Team
NIH
Biogen
Ionis Pharmaceuticals
PTC Therapeutics
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://www.jci.org/articles/view/124120/files/pdf
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5. Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1
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