Chemistry, structure and function of approved oligonucleotide therapeutics

M Egli, M Manoharan - Nucleic Acids Research, 2023 - academic.oup.com
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

microRNAs in action: biogenesis, function and regulation

R Shang, S Lee, G Senavirathne, EC Lai - Nature Reviews Genetics, 2023 - nature.com
Ever since microRNAs (miRNAs) were first recognized as an extensive gene family> 20
years ago, a broad community of researchers was drawn to investigate the universe of small …

Life of RISC: Formation, action, and degradation of RNA-induced silencing complex

H Iwakawa, Y Tomari - Molecular cell, 2022 - cell.com
Small RNAs regulate a wide variety of biological processes by repressing the expression of
target genes at the transcriptional and post-transcriptional levels. To achieve these …

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

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

Regulation of microRNA function in animals

LFR Gebert, IJ MacRae - Nature reviews Molecular cell biology, 2019 - nature.com
Since their serendipitous discovery in nematodes, microRNAs (miRNAs) have emerged as
key regulators of biological processes in animals. These small RNAs form complex networks …

[HTML][HTML] Metazoan micrornas

DP Bartel - Cell, 2018 - cell.com
MicroRNAs (miRNAs) are∼ 22 nt RNAs that direct posttranscriptional repression of mRNA
targets in diverse eukaryotic lineages. In humans and other mammals, these small RNAs …

Reexamining assumptions about miRNA-guided gene silencing

A Kilikevicius, G Meister, DR Corey - Nucleic acids research, 2022 - academic.oup.com
MicroRNAs (miRNAs) are short endogenously expressed RNAs that have the potential to
regulate the expression of any RNA. This potential has led to the publication of several …

RNA-targeted therapeutics

ST Crooke, JL Witztum, CF Bennett, BF Baker - Cell metabolism, 2018 - cell.com
RNA-targeted therapies represent a platform for drug discovery involving chemically
modified oligonucleotides, a wide range of cellular RNAs, and a novel target-binding motif …

Bridge RNAs direct programmable recombination of target and donor DNA

MG Durrant, NT Perry, JJ Pai, AR Jangid… - Nature, 2024 - nature.com
Genomic rearrangements, encompassing mutational changes in the genome such as
insertions, deletions or inversions, are essential for genetic diversity. These rearrangements …

The non-canonical aspects of microRNAs: many roads to gene regulation

CJ Stavast, SJ Erkeland - Cells, 2019 - mdpi.com
MicroRNAs (miRNAs) are critical regulators of gene expression. As miRNAs are frequently
deregulated in many human diseases, including cancer and immunological disorders, it is …