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dc.contributor.authorTang, Zhichao
dc.contributor.authorZhao, Junxing
dc.contributor.authorPearson, Zach J.
dc.contributor.authorBoskovic, Zarko V.
dc.contributor.authorWang, Jingxin
dc.date.accessioned2022-01-03T18:47:16Z
dc.date.available2022-01-03T18:47:16Z
dc.date.issued2021-04-14
dc.identifier.citationTang, Z.; Zhao, J.; Pearson, Z.J.; Boskovic, Z.V.; Wang, J. RNA-Targeting Splicing Modifiers: Drug Development and Screening Assays. Molecules 2021, 26, 2263. https://doi.org/10.3390/molecules26082263en_US
dc.identifier.urihttp://hdl.handle.net/1808/32314
dc.description.abstractRNA splicing is an essential step in producing mature messenger RNA (mRNA) and other RNA species. Harnessing RNA splicing modifiers as a new pharmacological modality is promising for the treatment of diseases caused by aberrant splicing. This drug modality can be used for infectious diseases by disrupting the splicing of essential pathogenic genes. Several antisense oligonucleotide splicing modifiers were approved by the U.S. Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Recently, a small-molecule splicing modifier, risdiplam, was also approved for the treatment of SMA, highlighting small molecules as important warheads in the arsenal for regulating RNA splicing. The cellular targets of these approved drugs are all mRNA precursors (pre-mRNAs) in human cells. The development of novel RNA-targeting splicing modifiers can not only expand the scope of drug targets to include many previously considered “undruggable” genes but also enrich the chemical-genetic toolbox for basic biomedical research. In this review, we summarized known splicing modifiers, screening methods for novel splicing modifiers, and the chemical space occupied by the small-molecule splicing modifiers.en_US
dc.publisherMDPIen_US
dc.rights© 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license.en_US
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/en_US
dc.subjectAlternative splicingen_US
dc.subjectHigh-throughput screeningen_US
dc.subjectAntisense oligonucleotideen_US
dc.subjectSmall moleculeen_US
dc.subjectSplicing modifieren_US
dc.subjectRNA-targetingen_US
dc.titleRNA-Targeting Splicing Modifiers: Drug Development and Screening Assaysen_US
dc.typeArticleen_US
kusw.kuauthorTang, Zhichao
kusw.kuauthorZhao, Junxing
kusw.kuauthorPearson, Zach J.
kusw.kuauthorBoskovic, Zarko V.
kusw.kuauthorWang, Jingxin
kusw.kudepartmentMedicinal Chemistryen_US
dc.identifier.doi10.3390/molecules26082263en_US
dc.identifier.orcidhttps://orcid.org/ 0000-0002-6305-6218en_US
dc.identifier.orcidhttps://orcid.org/ 0000-0001-9376-527Xen_US
dc.identifier.orcidhttps://orcid.org/ 0000-0002-9414-4093en_US
kusw.oaversionScholarly/refereed, publisher versionen_US
kusw.oapolicyThis item meets KU Open Access policy criteria.en_US
dc.identifier.pmidPMC8070285en_US
dc.rights.accessrightsopenAccessen_US


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© 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article
distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license.
Except where otherwise noted, this item's license is described as: © 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license.