Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation. [electronic resource]
- Human molecular genetics Feb 2009
- 546-55 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1460-2083
10.1093/hmg/ddn382 doi
Animals Base Sequence Exons Genetic Therapy Humans Mice Mice, Transgenic Molecular Sequence Data Muscular Atrophy, Spinal--genetics RNA Splicing RNA, Small Nuclear--genetics Survival of Motor Neuron 2 Protein--genetics