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Base editing rescue of spinal muscular atrophy in cells and in mice

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from SMN protein insufficiency following SMN1 loss. Approved therapies circumvent endogenous SMN regulation and require repeated dosing or may wane. We describe genome editing of SMN2, an insufficient copy of SMN1 h...

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Detalles Bibliográficos
Autores principales:	Arbab, Mandana, Matuszek, Zaneta, Kray, Kaitlyn M., Du, Ailing, Newby, Gregory A., Blatnik, Anton J., Raguram, Aditya, Richter, Michelle F., Zhao, Kevin T., Levy, Jonathan M., Shen, Max W., Arnold, W. David, Wang, Dan, Xie, Jun, Gao, Guangping, Burghes, Arthur H. M., Liu, David R.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10270003/ https://www.ncbi.nlm.nih.gov/pubmed/36996170 http://dx.doi.org/10.1126/science.adg6518

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