MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …

Antisense oligonucleotides (ASOs) interact with target RNAs via hybridization to modulate
gene expression through different mechanisms. ASO therapeutics are chemically modified …

Ionizable lipid nanoparticles (LNPs) have been widely used for in vivo delivery of RNA
therapeutics into the liver. However, a main challenge remains to develop LNP formulations …

Oligonucleotides (ONs) can interfere with biomolecules representing the entire extended
central dogma. Antisense gapmer, steric block, splice-switching ONs, and short interfering …

Fibrosis is the major determinant of morbidity and mortality in patients with nonalcoholic
steatohepatitis (NASH) but has no approved pharmacotherapy in part because of …

Chronic hepatitis B (CHB) is a major cause of liver-related morbidity and mortality.
Functional cure of CHB, defined as sustainable hepatitis B surface antigen (HBsAg) …

Antisense oligonucleotides (ASOs) are an increasingly represented class of drugs. These
small sequences of nucleotides are designed to precisely target other oligonucleotides …

Synthetic therapeutic oligonucleotides (STO) represent the third bonafide platform for drug
discovery in the pharmaceutical industry after small molecule and protein therapeutics. So …

Interactions of chemically modified nucleic acid therapeutics with plasma proteins play an
important role in facilitating distribution from the injection site to peripheral tissues by …

Therapeutic oligonucleotides are often modified using the phosphorothioate (PS) backbone
modification which enhances stability from nuclease mediated degradation. However …