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Diverse targets of SMN2-directed splicing-modulating small molecule therapeutics for spinal muscular atrophy

Designing an RNA-interacting molecule that displays high therapeutic efficacy while retaining specificity within a broad concentration range remains a challenging task. Risdiplam is an FDA-approved small molecule for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of infant...

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Detalles Bibliográficos
Autores principales:	Ottesen, Eric W, Singh, Natalia N, Luo, Diou, Kaas, Bailey, Gillette, Benjamin J, Seo, Joonbae, Jorgensen, Hannah J, Singh, Ravindra N
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2023
Materias:	Gene regulation, Chromatin and Epigenetics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10325915/ https://www.ncbi.nlm.nih.gov/pubmed/37026480 http://dx.doi.org/10.1093/nar/gkad259

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