Therapeutic siRNA: state of the art
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 …
against external invasion. It theoretically can silence any disease-related genes in a …
Recent progress in non-native nucleic acid modifications
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 …
inherent programmability, synthetic accessibility and wide functionality of these nucleic acids …
Ongoing clinical trials of nonviral siRNA therapeutics
Short interfering RNAs (siRNA) are a powerful class of genetic medicines whose clinical
translation can be hindered by their suboptimal delivery properties in vivo. Here, we provide …
translation can be hindered by their suboptimal delivery properties in vivo. Here, we provide …
Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs
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 …
can be recognized by complementary base pairing, synthetic oligonucleotides and …
The medicinal chemistry of therapeutic oligonucleotides
WB Wan, PP Seth - Journal of medicinal chemistry, 2016 - ACS Publications
Oligonucleotide-based therapeutics have made rapid progress in the clinic for treatment of a
variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules …
variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules …
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
TP Prakash, MJ Graham, J Yu, R Carty… - Nucleic acids …, 2014 - academic.oup.com
Triantennary N-acetyl galactosamine (GalNAc, GN3), a high-affinity ligand for the hepatocyte-
specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation …
specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation …
A single-cell map of antisense oligonucleotide activity in the brain
MA Mortberg, JE Gentile, NM Nadaf… - Nucleic Acids …, 2023 - academic.oup.com
Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …
STAT3 antisense oligonucleotide AZD9150 in a subset of patients with heavily pretreated lymphoma: results of a phase 1b trial
MJ Reilley, P McCoon, C Cook, P Lyne… - … for immunotherapy of …, 2018 - Springer
Abstract Background The Janus kinase (JAK) and signal transduction and activation of
transcription (STAT) signaling pathway is an attractive target in multiple cancers. Activation …
transcription (STAT) signaling pathway is an attractive target in multiple cancers. Activation …
The interaction of phosphorothioate-containing RNA targeted drugs with proteins is a critical determinant of the therapeutic effects of these agents
Recent progress in understanding phosphorothioate antisense oligonucleotide (PS-ASO)
interactions with proteins has revealed that proteins play deterministic roles in the …
interactions with proteins has revealed that proteins play deterministic roles in the …
Pharmacology of a central nervous system delivered 2′-O-methoxyethyl–modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
F Rigo, SJ Chun, DA Norris, G Hung, S Lee… - … of Pharmacology and …, 2014 - ASPET
Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease caused by the loss
of survival of motor neuron (SMN) protein. Previously, we demonstrated that ISIS 396443, an …
of survival of motor neuron (SMN) protein. Previously, we demonstrated that ISIS 396443, an …