MicroRNA-based diagnosis and therapy

PTB Ho, IM Clark, LTT Le - International journal of molecular sciences, 2022 - mdpi.com
MicroRNAs (miRNAs) are a group of endogenous non-coding RNAs that regulate gene
expression. Alteration in miRNA expression results in changes in the profile of genes …

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 …

[HTML][HTML] Innovative approaches for cancer treatment: Current perspectives and new challenges

C Pucci, C Martinelli, G Ciofani - ecancermedicalscience, 2019 - ncbi.nlm.nih.gov
Every year, cancer is responsible for millions of deaths worldwide and, even though much
progress has been achieved in medicine, there are still many issues that must be addressed …

DNA repair pathway choices in CRISPR-Cas9-mediated genome editing

C Xue, EC Greene - Trends in Genetics, 2021 - cell.com
Many clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-
associated protein 9 (Cas9)-based genome editing technologies take advantage of Cas …

TLR7 gain-of-function genetic variation causes human lupus

GJ Brown, PF Cañete, H Wang, A Medhavy, J Bones… - Nature, 2022 - nature.com
Although circumstantial evidence supports enhanced Toll-like receptor 7 (TLR7) signalling
as a mechanism of human systemic autoimmune disease,,,,,–, evidence of lupus-causing …

Knocking down disease: a progress report on siRNA therapeutics

A Wittrup, J Lieberman - Nature Reviews Genetics, 2015 - nature.com
Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence
complementarity, can be used therapeutically to block the synthesis of disease-causing …

Discriminating self from non-self in nucleic acid sensing

M Schlee, G Hartmann - Nature Reviews Immunology, 2016 - nature.com
Innate immunity against pathogens relies on an array of immune receptors to detect
molecular patterns that are characteristic of the pathogens, including receptors that are …

[HTML][HTML] Huntingtin lowering strategies for disease modification in Huntington's disease

SJ Tabrizi, R Ghosh, BR Leavitt - Neuron, 2019 - cell.com
Huntington's disease is caused by an abnormally expanded CAG repeat expansion in the
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …

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 …