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Viltolarsen (Synonyms: NS-065/NCNP-01)

Cat. No.: HY-132586
Handling Instructions

Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD).

For research use only. We do not sell to patients.

Viltolarsen Chemical Structure

Viltolarsen Chemical Structure

CAS No. : 2055732-84-6

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Description

Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD)[1].

In Vitro

Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has emerged as a promising therapy for DMD. Antisense oligonucleotides (AONs) are designed to induce the skipping of exon(s), in order to restore the reading frame, and therefore, allow for dystrophin expression[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

CAS No.
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Viltolarsen
Cat. No.:
HY-132586
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