The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …

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 …

Genetic robustness, or the ability of an organism to maintain fitness in the presence of
harmful mutations, can be achieved via protein feedback loops. Previous work has …

Recent advancements in the production, modification, and cellular delivery of RNA
molecules facilitated the expansion of RNA-based therapeutics. The increasing …

Several recent studies in a number of model systems including zebrafish, Arabidopsis, and
mouse have revealed phenotypic differences between knockouts (ie, mutants) and …

Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

MicroRNAs (miRNAs) are endogenous non-coding RNAs that contain approximately 22
nucleotides. They serve as key regulators in various biological processes and their …

Functional nucleic acid (NA)-based drugs have a broad range of applications since they
allow the alteration and control of gene/protein expression patterns in cells. In principle …

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