Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. All patients have at least one copy of a paralog, SMN2, but a C-to-T transition in this gene results in exon 7 skipping in a majority of transcripts. Approved treatment for SMA i...

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Detalles Bibliográficos
Autores principales: Winkelsas, Audrey M., Grunseich, Christopher, Harmison, George G., Chwalenia, Katarzyna, Rinaldi, Carlo, Hammond, Suzan M., Johnson, Kory, Bowerman, Melissa, Arya, Sukrat, Talbot, Kevin, Wood, Matthew J., Fischbeck, Kenneth H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7851419/
https://www.ncbi.nlm.nih.gov/pubmed/33575118
http://dx.doi.org/10.1016/j.omtn.2020.12.027

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