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 …

The rapid expansion of the available genomic data continues to greatly impact biomedical
science and medicine. Fulfilling the clinical potential of genetic discoveries requires the …

Small-molecule and protein/antibody drugs mainly act on genome-derived proteins to exert
pharmacological effects. RNA based therapies hold the promise to expand the range of …

A recent revolution in RNA biology has led to the identification of new RNA classes with
unanticipated functions, new types of RNA modifications, an unexpected multiplicity of …

The concepts of developing RNAs as new molecular entities for therapies have arisen again
and again since the discoveries of antisense RNAs, direct RNA-protein interactions …

Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

The therapeutic pathways that modulate transcription mechanisms currently include gene
knockdown and splicing modulation. However, additional mechanisms may come into play …

Recent advances in understanding different RNAs and unique features of their biology have
revealed a wealth of information. However, approaches to identify small molecules that …

Ribonucleic acid (RNA) therapeutics are being actively researched as a therapeutic
modality in preclinical and clinical studies. They have become one of the most ubiquitously …

Since the characterization of mRNA in 1961, our understanding of the roles of RNA
molecules has significantly grown. Beyond serving as a link between DNA and proteins …