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].
Molecular Weight:
6924.80
Purity:
95.25
CAS Number:
[2055732-84-6]
Formula:
C244H381N113O88P20
Target:
Dystrophin,Nucleoside Antimetabolite/Analog
Application Notes:
Oligonucleotides
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