RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

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

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

Multicomponent deoxyribozymes (MNAzymes) have great potential in gene therapy, but
their ability to recognize disease tissue and further achieve synergistic gene regulation has …

Background The glucagon-like peptide-1 (GLP-1) is a multifaceted hormone with broad
pharmacological potential. Among the numerous metabolic effects of GLP-1 are the glucose …

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 …

Antisense technology is beginning to deliver on the broad promise of the technology. Ten
RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double …

Diabetes mellitus is characterized by the failure of insulin-secreting pancreatic β-cells (or β-
cell death) due to either autoimmunity (type 1 diabetes mellitus) or failure to compensate for …

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

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