Rescue of spinal muscular atrophy mouse models with AAV9-Exon-specific U1 snRNA

Spinal Muscular Atrophy results from loss-of-function mutations in SMN1 but correcting aberrant splicing of SMN2 offers hope of a cure. However, current splice therapy requires repeated infusions and is expensive. We previously rescued SMA mice by promoting the inclusion of a defective exon in SMN2...

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Dades bibliogràfiques
Publicat a:Nucleic Acids Res
Autors principals: Donadon, Irving, Bussani, Erica, Riccardi, Federico, Licastro, Danilo, Romano, Giulia, Pianigiani, Giulia, Pinotti, Mirko, Konstantinova, Pavlina, Evers, Melvin, Lin, Shuo, Rüegg, Markus A, Pagani, Franco
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2019
Matèries:
RNA and RNA-protein complexes
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6698663/
https://ncbi.nlm.nih.gov/pubmed/31127278
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz469
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