Target Gene Table
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TCGA Plot Options
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Drug Information
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| Gene | DMD |
|---|---|
| DrugBank ID | DB15593 |
| Drug Name | Golodirsen |
| Target ID | BE0009794 |
| UniProt ID | P11532 |
| Regulation Type | inducer |
| PubMed IDs | 29301272; 24554202; 30171533; 31576784 |
| Citations | Aslesh T, Maruyama R, Yokota T: Skipping Multiple Exons to Treat DMD-Promises and Challenges. Biomedicines. 2018 Jan 2;6(1). pii: biomedicines6010001. doi: 10.3390/biomedicines6010001.@@Kinter J, Sinnreich M: Molecular targets to treat muscular dystrophies. Swiss Med Wkly. 2014 Feb 19;144:w13916. doi: 10.4414/smw.2014.13916.@@Rodrigues M, Yokota T: An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases. Methods Mol Biol. 2018;1828:31-55. doi: 10.1007/978-1-4939-8651-4_2.@@Hwang J, Yokota T: Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies. Expert Rev Mol Med. 2019 Oct 2;21:e5. doi: 10.1017/erm.2019.5. |
| Groups | Approved |
| Direct Classification | |
| SMILES | |
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| ChEMBL |
