| Chemical Properties | Back Directory | [storage temp. ]
Store at -20°C | [form ]
Solid | [color ]
White to off-white | [Sequence]
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C) |
| Hazard Information | Back Directory | [Uses]
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]. | [References]
[1] Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. DOI:10.1007/s40265-020-01339-3
[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. DOI:10.1016/j.omtn.2018.09.017 |
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| Company Name: |
Apextide Co Ltd Gold
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| Tel: |
15300650552 |
| Website: |
www.apextide.com |
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