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Article

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

Nucleic acids research - 20 Aug 2021

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

Abstract excerpt

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 binding site. Importantly, the sequence of this GA-rich binding site significantly enhanced the potency of risdiplam...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 34283224
DOI: 10.1093/nar/gkab602
PMCID: PMC8373063

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