Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide designed to target dystrophin mRNA by binding to exon 53. This mechanism promotes exon skipping, leading to the restoration of the open-reading frame and the production of a truncated dystrophin protein that preserves essential functional elements. Viltolarsen is primarily utilized in research related to Duchenne muscular dystrophy (DMD), making it a valuable tool for advancing therapeutic strategies in this condition.
Viltolarsen is a phosphorodiamidate morpholino antisense oligonucleotide designed to target dystrophin mRNA by binding to exon 53. This mechanism promotes exon skipping, leading to the restoration of the open-reading frame and the production of a truncated dystrophin protein that preserves essential functional elements. Viltolarsen is primarily utilized in research related to Duchenne muscular dystrophy (DMD), making it a valuable tool for advancing therapeutic strategies in this condition.
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