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 …

RNA drugs and RNA targets for small molecules: principles, progress, and challenges

AM Yu, YH Choi, MJ Tu, RM TOUYZ - Pharmacological reviews, 2020 - Elsevier
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

A lysosome‐activated tetrahedral nanobox for encapsulated siRNA delivery

Y Gao, X Chen, T Tian, T Zhang, S Gao… - Advanced …, 2022 - Wiley Online Library
Tetrahedral framework nucleic acids (tFNAs) have attracted extensive attention as drug
nanocarriers because of their excellent cellular uptake. However, for oligonucleotide cargos …

SARS-CoV-2-mediated dysregulation of metabolism and autophagy uncovers host-targeting antivirals

NC Gassen, J Papies, T Bajaj, J Emanuel… - Nature …, 2021 - nature.com
Viruses manipulate cellular metabolism and macromolecule recycling processes like
autophagy. Dysregulated metabolism might lead to excessive inflammatory and …

The current state and future directions of RNAi-based therapeutics

RL Setten, JJ Rossi, S Han - Nature reviews Drug discovery, 2019 - nature.com
The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all
human cells. Small double-stranded RNA molecules can efficiently trigger RNAi silencing of …

Structure of the human DICER–pre-miRNA complex in a dicing state

YY Lee, H Lee, H Kim, VN Kim, SH Roh - Nature, 2023 - nature.com
Dicer has a key role in small RNA biogenesis, processing double-stranded RNAs
(dsRNAs),. Human DICER (hDICER, also known as DICER1) is specialized for cleaving …

siRNA versus miRNA as therapeutics for gene silencing

JKW Lam, MYT Chow, Y Zhang, SWS Leung - Molecular Therapy-Nucleic …, 2015 - cell.com
Discovered a little over two decades ago, small interfering RNAs (siRNAs) and microRNAs
(miRNAs) are noncoding RNAs with important roles in gene regulation. They have recently …

Overcoming barriers for siRNA therapeutics: from bench to bedside

MI Sajid, M Moazzam, S Kato, K Yeseom Cho… - Pharmaceuticals, 2020 - mdpi.com
The RNA interference (RNAi) pathway possesses immense potential in silencing any gene
in human cells. Small interfering RNA (siRNA) can efficiently trigger RNAi silencing of …

Sequence determinant of small RNA production by DICER

YY Lee, H Kim, VN Kim - Nature, 2023 - nature.com
RNA silencing relies on specific and efficient processing of double-stranded RNA by Dicer,
which yields microRNAs (miRNAs) and small interfering RNAs (siRNAs),. However, our …

RNA interference in the era of nucleic acid therapeutics

V Jadhav, A Vaishnaw, K Fitzgerald, MA Maier - Nature Biotechnology, 2024 - nature.com
Two decades of research on RNA interference (RNAi) have transformed a breakthrough
discovery in biology into a robust platform for a new class of medicines that modulate mRNA …