Base editing as a genetic treatment for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a devastating neuromuscular disease caused by mutations in the SMN1 gene. Despite the development of various therapies, outcomes can remain suboptimal in SMA infants and the duration of such therapies are uncertain. SMN2 is a paralogous gene that mainly differs from...

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Detalles Bibliográficos
Autores principales: Alves, Christiano R. R., Ha, Leillani L., Yaworski, Rebecca, Lazzarotto, Cicera R., Christie, Kathleen A., Reilly, Aoife, Beauvais, Ariane, Doll, Roman M., de la Cruz, Demitri, Maguire, Casey A., Swoboda, Kathryn J., Tsai, Shengdar Q., Kothary, Rashmi, Kleinstiver, Benjamin P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9882371/
https://www.ncbi.nlm.nih.gov/pubmed/36711797
http://dx.doi.org/10.1101/2023.01.20.524978

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