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.
In biology, a variant refers to a specific region of the genome that differs between two genomes, or an individual organism with characteristics that deviate from the standard within a species.
A transcription factor is a protein that regulates the transcription of genetic information from DNA to RNA, playing a pivotal role in the regulation of gene expression.