Non‐coding RNAs: regulators of disease

RJ Taft, KC Pang, TR Mercer, M Dinger… - The Journal of …, 2010 - Wiley Online Library
For 50 years the term 'gene'has been synonymous with regions of the genome encoding
mRNAs that are translated into protein. However, recent genome‐wide studies have shown …

Tapping the RNA world for therapeutics

J Lieberman - Nature structural & molecular biology, 2018 - nature.com
A recent revolution in RNA biology has led to the identification of new RNA classes with
unanticipated functions, new types of RNA modifications, an unexpected multiplicity of …

[HTML][HTML] Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells

HK Liao, Y Gu, A Diaz, J Marlett, Y Takahashi… - Nature …, 2015 - nature.com
To combat hostile viruses, bacteria and archaea have evolved a unique antiviral defense
system composed of clustered regularly interspaced short palindromic repeats (CRISPRs) …

Lentivirus-delivered stable gene silencing by RNAi in primary cells

SA Stewart, DM Dykxhoorn, D Palliser, H Mizuno… - Rna, 2003 - rnajournal.cshlp.org
Genome-wide genetic approaches have proven useful for examining pathways of biological
significance in model organisms such as Saccharomyces cerevisiae, Drosophila …

[HTML][HTML] Single-stranded antisense siRNAs guide target RNA cleavage in RNAi

J Martinez, A Patkaniowska, H Urlaub, R Lührmann… - Cell, 2002 - cell.com
Small interfering RNAs (siRNAs) are the mediators of mRNA degradation in the process of
RNA interference (RNAi). Here, we describe a human biochemical system that recapitulates …

Gene silencing in mammals by small interfering RNAs

MT McManus, PA Sharp - Nature reviews genetics, 2002 - nature.com
Among the 3 billion base pairs of the human genome, there are∼ 30,000–40,000 protein-
coding genes, but the function of at least half of them remains unknown. A new tool—short …

Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies

Y Deng, CC Wang, KW Choy, Q Du, J Chen, Q Wang… - Gene, 2014 - Elsevier
During recent decades there have been remarkable advances in biology, in which one of
the most important discoveries is RNA interference (RNAi). RNAi is a specific post …

RNAi therapeutics: principles, prospects and challenges

L Aagaard, JJ Rossi - Advanced drug delivery reviews, 2007 - Elsevier
RNA interference (RNAi) was discovered less than a decade ago and already there are
human clinical trials in progress or planned. A major advantage of RNAi versus other …

Strategies for silencing human disease using RNA interference

DH Kim, JJ Rossi - Nature Reviews Genetics, 2007 - nature.com
Since the first description of RNA interference (RNAi) in animals less than a decade ago,
there has been rapid progress towards its use as a therapeutic modality against human …

siRNA delivery systems for cancer treatment

YK Oh, TG Park - Advanced drug delivery reviews, 2009 - Elsevier
With increasing knowledge on the molecular mechanisms of endogenous RNA interference,
small interfering RNAs (siRNAs) have been emerging as innovative nucleic acid medicines …