Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium 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 sodium has the potential for Duchenne muscular dystrophy (DMD) research[1][2].
Molekulargewicht:
6924.80
Reinheit:
95.25
Formel:
C244H360N113Na21O88P20
Target-Kategorie:
Dystrophin,Nucleoside Antimetabolite/Analog
Anwendungsbeschreibung:
Oligonucleotides
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