Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. [electronic resource]
Producer: 20090714Description: 1600-11 p. digitalISSN:- 1460-2083
- Animals
- Cells, Cultured
- Disease Models, Animal
- Fibroblasts -- metabolism
- Genetic Therapy
- Humans
- Introns
- Mice
- Mice, Transgenic
- Muscular Atrophy, Spinal -- genetics
- RNA, Antisense -- chemistry
- RNA-Binding Proteins -- genetics
- Regulatory Sequences, Nucleic Acid
- Survival of Motor Neuron 2 Protein -- genetics
No physical items for this record
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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