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Recognition of single-stranded nucleic acids by small-molecule splicing modulators

Risdiplam is the first approved small-molecule splicing modulator for the treatment of spinal muscular atrophy (SMA). Previous studies demonstrated that risdiplam analogues have two separate binding sites in exon 7 of the SMN2 pre-mRNA: (i) the 5′-splice site and (ii) an upstream purine (GA)-rich bi...

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Detalles Bibliográficos
Autores principales:	Tang, Zhichao, Akhter, Sana, Ramprasad, Ankita, Wang, Xiao, Reibarkh, Mikhail, Wang, Jinan, Aryal, Sadikshya, Thota, Srinivas S, Zhao, Junxing, Douglas, Justin T, Gao, Philip, Holmstrom, Erik D, Miao, Yinglong, Wang, Jingxin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	Chemical Biology and Nucleic Acid Chemistry
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8373063/ https://www.ncbi.nlm.nih.gov/pubmed/34283224 http://dx.doi.org/10.1093/nar/gkab602

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