More than 25 years after its discovery, the post-transcriptional gene regulation mechanism
termed RNAi is now transforming pharmaceutical development, proved by the recent FDA …

RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all
human cells. Small double-stranded RNA molecules can efficiently trigger RNAi silencing of …

Animal models play an indispensable role in the study of human diseases. However, animal
models of different diseases do not fully mimic the complex internal environment of humans …

The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in
1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was …

The oligonucleotide therapeutics field has seen remarkable progress over the last few years
with the approval of the first antisense drug and with promising developments in late stage …

Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence
complementarity, can be used therapeutically to block the synthesis of disease-causing …

MicroRNAs (miRNAs) are evolutionarily conserved small non-coding RNAs that have crucial
roles in regulating gene expression. Increasing evidence supports a role for miRNAs in …

Nanosystems have shown encouraging outcomes and substantial progress in the areas of
drug delivery and biomedical applications. However, the controlled and targeted delivery of …