Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
2010
Spinal muscular atrophy is an autosomal recessive disease of motor neurons caused by lack of the SMN gene. Foust et al. achieve long-term correction of the disease phenotype in a mouse model by intravenous delivery of SMN using the viral vector scAAV9.
Keywords:
- Correction
- Source
- Cite
- Save
- Machine Reading By IdeaReader
29
References
576
Citations
NaN
KQI