Antisense technology is beginning to deliver on the broad promise of the technology. Ten RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double-strand ASOs (siRNAs) have now been approved for commercial use, and the ASOs in phase 2/3 trials are innovative, delivered by multiple routes of administration and
The most commonly used Cas9 nuclease, derived from S. pyogenes, recognizes a PAM sequence of NGG that is found directly downstream of the target sequence in the genomic DNA, on the non-target strand. Recognition of the PAM by the Cas9 nuclease is thought to destabilize the adjacent sequence, allowing interrogation of the sequence by the crRNAGene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. [1] [2] Gene silencing can occur during either transcription or translation and is often used in research. [1] [2] In particular, methods used to silence genes are being increasingly used to produce therapeutics to combat cancer and other
Intronic L1 sequences distribute either in the same or reverse direction as the hosting genes (i.e., sense vs antisense), at a ratio of approximately 1:2 (Fig. 1c). CpG DNA as TLR agonist is required at mg/kg doses for immune stimulation, whereas antisense and siRNA agents are required at mg/kg quantitites for gene silencing activity. RmShC. 376 89 399 136 344 441 470 0 219