Aptamers are oligonucleotide sequences with a length of about 25–80 bases which have
abilities to bind to specific target molecules that rival those of monoclonal antibodies. They …

After nearly 40 years of development, oligonucleotide therapeutics are nearing meaningful
clinical productivity. One of the key advantages of oligonucleotide drugs is that their delivery …

Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide
variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease …

RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs),
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …

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 …

Targeted delivery of oligonucleotides to liver hepatocytes using N-acetylgalactosamine
(GalNAc) conjugates that bind to the asialoglycoprotein receptor has become a …

Abstract Lipoprotein (a)[Lp (a)] has risen to the level of an accepted cardiovascular disease
risk factor, but final proof of causality awaits a randomized trial of Lp (a) lowering. Inhibiting …

Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …

Abstract Aims Elevated apolipoprotein C-III (apoC-III) levels are associated with
hypertriglyceridaemia and coronary heart disease. AKCEA-APOCIII-LRx is an N-acetyl …

A main challenge in realizing the full potential of nucleic acid therapeutics is efficient
delivery of them into targeted tissues and cells. N-acetylgalactosamine (GalNAc) is a well …