2055732-84-6
Chemical Structure
Viltolarsen
Synonym(s): NS-065/NCNP-01
- CAS 番号: 2055732-84-6
- Formula:C244H381N113O88P20
- Molecular Weight:6924.82
SMILES: OC[C@H]1O[C@@H](n(ccc(N)n2)c2=O)CN(P(N(C)C)(OC[C@H]3O[C@@H](n(ccc(N)n4)c4=O)CN(P(N(C)C)(OC[C@H]5O[C@@H](n(cc(C)c(=O)[nH]6)c6=O)CN(P(N(C)C)(OC[C@H]7O[C@@H](n(ccc(N)n8)c8=O)CN(P(N(C)C)(OC[C@H]9O[C@@H](n(ccc(N)n%10)c%10=O)CN(P(N(C)C)(OC[C@H]%11O[C@@H](n(cnc%12%13)c%12nc(N)[nH]c%13=O)CN(P(N(C)C)(OC[C@H]%14O[C@@H](n(cnc%15%16)c%15nc(N)[nH]c%16=O)CN(P(N(C)C)(OC[C@H]%17O[C@@H](n(cc(C)c(=O)[nH]%18)c%18=O)CN(P(N(C)C)(OC[C@H]%19O[C@@H](n(cc(C)c(=O)[nH]%20)c%20=O)CN(P(N(C)C)(OC[C@@H]%21CN(P(N(C)C)(OC[C@H]%22O[C@@H](n(cc(C)c(=O)[nH]%23)c%23=O)CN(P(N(C)C)(OC[C@H]%24O[C@@H](n(cnc%25%26)c%25nc(N)[nH]c%26=O)CN(P(N(C)C)(OC[C@H]%27O[C@@H](n%28c%29ncnc(N)c%29nc%28)CN(P(N(C)C)(OC[C@H]%30O[C@@H](n%31c%32ncnc(N)c%32nc%31)CN(P(N(C)C)(OC[C@H]%33O[C@@H](n(cnc%34%35)c%34nc(N)[nH]c%35=O)CN(P(N(C)C)(OC[C@H]%36O[C@@H](n(cnc%37%38)c%37nc(N)[nH]c%38=O)CN(P(N(C)C)(OC[C@H]%39O[C@@H](n(cc(C)c(=O)[nH]%40)c%40=O)CN(P(N(C)C)(OC[C@H]%41O[C@@H](n(cnc%42%43)c%42nc(N)[nH]c%43=O)CN(P(N(C)C)(OC[C@H]%44O[C@@H](n(cc(C)c(=O)[nH]%45)c%45=O)CN(P(N(C)C)(OC[C@H]%46O[C@@H](n(cc(C)c(=O)[nH]%47)c%47=O)CN(P(N(C)C)(OC[C@@H]%48CNC[C@H](n(ccc(N)n%49)c%49=O)O%48)=O)C%46)=O)C%44)=O)C%41)=O)C%39)=O)C%36)=O)C%33)=O)C%30)=O)C%27)=O)C%24)=O)C%22)=O)C[C@H](n(ccc(N)n%50)c%50=O)O%21)=O)C%19)=O)C%17)=O)C%14)=O)C%11)=O)C9)=O)C7)=O)C5)=O)C3)=O)C1
Biological Activity: Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research[1][2].
| 製品番号 | 製品名 | 純度 | 製品説明 | Pricing | |||||||||||||||||||
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Viltolarsen | 95.25% | Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research. | ||||||||||||||||||||
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- [1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. [Content Brief]
- [2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449. [Content Brief]
Keywords
