RNA-based therapeutics: an overview and prospectus

Y Zhu, L Zhu, X Wang, H Jin - Cell death & disease, 2022 - nature.com
The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

Noncoding RNA therapeutics—challenges and potential solutions

M Winkle, SM El-Daly, M Fabbri, GA Calin - Nature reviews Drug …, 2021 - nature.com
Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …

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 …

Crossing the blood-brain barrier: advances in nanoparticle technology for drug delivery in neuro-oncology

AM Hersh, S Alomari, BM Tyler - International journal of molecular …, 2022 - mdpi.com
The blood-brain barrier (BBB) constitutes a microvascular network responsible for excluding
most drugs from the brain. Treatment of brain tumors is limited by the impermeability of the …

[HTML][HTML] Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

S Zhang, J Shen, D Li, Y Cheng - Theranostics, 2021 - ncbi.nlm.nih.gov
CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Nucleic acid delivery for therapeutic applications

A Gupta, JL Andresen, RS Manan, R Langer - Advanced Drug Delivery …, 2021 - Elsevier
Recent medical advances have exploited the ability to address a given disease at the
underlying level of transcription and translation. These treatment paradigms utilize nucleic …

Nanoagent‐promoted mild‐temperature photothermal therapy for cancer treatment

G Gao, X Sun, G Liang - Advanced Functional Materials, 2021 - Wiley Online Library
Photothermal therapy (PTT), which utilizes near‐infrared light‐absorbing agents to ablate
tumor, has emerged as a highly promising anticancer strategy and received intensive …

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 …

A historical review of brain drug delivery

WM Pardridge - Pharmaceutics, 2022 - mdpi.com
The history of brain drug delivery is reviewed beginning with the first demonstration, in 1914,
that a drug for syphilis, salvarsan, did not enter the brain, due to the presence of a blood …