[HTML][HTML] Emerging concepts of miRNA therapeutics: from cells to clinic
MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …
Noncoding RNA therapeutics—challenges and potential solutions
Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …
[HTML][HTML] Programming inactive RNA-binding small molecules into bioactive degraders
Target occupancy is often insufficient to elicit biological activity, particularly for RNA,
compounded by the longstanding challenges surrounding the molecular recognition of RNA …
compounded by the longstanding challenges surrounding the molecular recognition of RNA …
Targeting RNA with small molecules: from fundamental principles towards the clinic
Recent advances in our understanding of RNA biology have uncovered crucial roles for
RNA in multiple disease states, ranging from viral and bacterial infections to cancer and …
RNA in multiple disease states, ranging from viral and bacterial infections to cancer and …
Amplifying gene expression with RNA-targeted therapeutics
O Khorkova, J Stahl, A Joji, CH Volmar… - Nature Reviews Drug …, 2023 - nature.com
Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …
from increased expression of the corresponding protein. However, in drug development, it …
Targeting Xist with compounds that disrupt RNA structure and X inactivation
Although more than 98% of the human genome is non-coding, nearly all of the drugs on the
market target one of about 700 disease-related proteins. The historical reluctance to invest …
market target one of about 700 disease-related proteins. The historical reluctance to invest …
Non-coding RNAs in cancer: Platforms and strategies for investigating the genomic “dark matter”
The discovery of the role of non-coding RNAs (ncRNAs) in the onset and progression of
malignancies is a promising frontier of cancer genetics. It is clear that ncRNAs are …
malignancies is a promising frontier of cancer genetics. It is clear that ncRNAs are …
Small molecule recognition of disease-relevant RNA structures
SM Meyer, CC Williams, Y Akahori, T Tanaka… - Chemical Society …, 2020 - pubs.rsc.org
Targeting RNAs with small molecules represents a new frontier in drug discovery and
development. The rich structural diversity of folded RNAs offers a nearly unlimited reservoir …
development. The rich structural diversity of folded RNAs offers a nearly unlimited reservoir …
Targeting RNA with small molecules to capture opportunities at the intersection of chemistry, biology, and medicine
MD Disney - Journal of the American Chemical Society, 2019 - ACS Publications
The biology of healthy and disease-affected cells is often mediated by RNA structures,
desirable targets for small molecule chemical probes and lead medicines. Although …
desirable targets for small molecule chemical probes and lead medicines. Although …
How we think about targeting RNA with small molecules
MG Costales, JL Childs-Disney, HS Haniff… - Journal of medicinal …, 2020 - ACS Publications
RNA offers nearly unlimited potential as a target for small molecule chemical probes and
lead medicines. Many RNAs fold into structures that can be selectively targeted with small …
lead medicines. Many RNAs fold into structures that can be selectively targeted with small …