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Pack Size | Price | Availability | Quantity |
---|---|---|---|
1 mg | $997 | Backorder | |
5 mg | $2,550 | Backorder | |
10 mg | $3,990 | Backorder |
Description | Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically targets the splicing of exon 53 in the dystrophin gene. It is utilized in research related to Duchenne muscular dystrophy (DMD). |
In vitro | Duchenne muscular dystrophy (DMD), a prevalent and fatal muscle-degenerative condition affecting young boys, is caused by mutations in the DMD gene. Exon skipping has gained recognition as a potential treatment strategy for DMD. Antisense oligonucleotides (AONs) are engineered to promote the omission of specific exon(s), aiming to rectify the reading frame and consequently enable the production of dystrophin[1]. |
Alias | NS-065, NCNP-01 |
Cas No. | 2055732-84-6 |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
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