The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

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

Evidence accumulated over the past decade shows that long non-coding RNAs (lncRNAs)
are widely expressed and have key roles in gene regulation. Recent studies have begun to …

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

ABSTRACT RNA interference (RNAi) is an ancient biological mechanism used to defend
against external invasion. It theoretically can silence any disease-related genes in a …

Nucleic acid‐based therapeutics that regulate gene expression have been developed
towards clinical use at a steady pace for several decades, but in recent years the field has …

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 …

While Nature harnesses RNA and DNA to store, read and write genetic information, the
inherent programmability, synthetic accessibility and wide functionality of these nucleic acids …

Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

RNA plays a central role in the expression of all genes. Because any sequence within RNA
can be recognized by complementary base pairing, synthetic oligonucleotides and …