Antisense technology: an overview and prospectus

ST Crooke, BF Baker, RM Crooke… - Nature reviews Drug …, 2021 - nature.com
Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …

Advances in oligonucleotide drug delivery

TC Roberts, R Langer, MJA Wood - Nature reviews Drug discovery, 2020 - nature.com
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Therapeutic siRNA: state of the art

B Hu, L Zhong, Y Weng, L Peng, Y Huang… - Signal transduction and …, 2020 - nature.com
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 …

Antisense technology: A review

ST Crooke, XH Liang, BF Baker, RM Crooke - Journal of Biological …, 2021 - ASBMB
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 …

Overcoming cellular barriers for RNA therapeutics

SF Dowdy - Nature biotechnology, 2017 - nature.com
RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs),
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …

The delivery of therapeutic oligonucleotides

RL Juliano - Nucleic acids research, 2016 - academic.oup.com
The oligonucleotide therapeutics field has seen remarkable progress over the last few years
with the approval of the first antisense drug and with promising developments in late stage …

Phosphorothioate modified oligonucleotide–protein interactions

ST Crooke, TA Vickers, X Liang - Nucleic acids research, 2020 - academic.oup.com
Antisense oligonucleotides (ASOs) interact with target RNAs via hybridization to modulate
gene expression through different mechanisms. ASO therapeutics are chemically modified …

Cellular uptake and trafficking of antisense oligonucleotides

ST Crooke, S Wang, TA Vickers, W Shen… - Nature …, 2017 - nature.com
Antisense oligonucleotides (ASOs) modified with phosphorothioate (PS) linkages and
different 2′ modifications can be used either as drugs (eg, to treat homozygous familial …

Therapeutic oligonucleotides: state of the art

CIE Smith, R Zain - Annual review of pharmacology and …, 2019 - annualreviews.org
Oligonucleotides (ONs) can interfere with biomolecules representing the entire extended
central dogma. Antisense gapmer, steric block, splice-switching ONs, and short interfering …

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

W Shen, CL De Hoyos, MT Migawa, TA Vickers… - Nature …, 2019 - nature.com
The molecular mechanisms of toxicity of chemically modified phosphorothioate antisense
oligonucleotides (PS-ASOs) are not fully understood. Here, we report that toxic gapmer PS …