Golodirsen is a phosphorodiamidate morpholino oligomer (PMO), a class of synthetic oligonucleotides designed for high stability and resistance to enzymatic degradation.
siRNA and ASO are two nucleic acid-based gene silencing technologies that achieve precise regulation of gene expression by complementary pairing with specific mRNA sequences.
RNA Interference (RNAi) is a gene-silencing mechanism first discovered in Caenorhabditis elegans, triggered by double-stranded small interfering RNA (siRNA).
The development and application of siRNA drugs mark a revolution in the field of gene therapy, as they cleverly utilize the cellular RNA interference (RNAi) pathway to achieve precise regulation of disease-related gene expression.
Patsnap Bio, with its chemical modification search function, provides users with a powerful tool for exploring multidimensional nucleotide sequence information.