Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
Cold Spring Harbor Laboratory · Ionis Pharmaceuticals (United States)
Indexed incrossrefpubmed
Abstract
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Citation impact
740
total citations
- FWCI
- 25.93
- Percentile
- 100%
- References
- 31
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Authors
7Topics & keywords
Topics
Keywords
- SMN1
- Spinal muscular atrophy
- SMA*
- Motor neuron
- Spinal cord
- Biology
- Neuroscience
- Alternative splicing
UN Sustainable Development Goals
- Good health and well-being
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