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Progress towards the development of the small molecule equivalent of small interfering RNA (siRNA)
- Source :
- Curr Opin Chem Biol
- Publication Year :
- 2020
-
Abstract
- Given that many small molecules could bind to structured regions at sites that will not affect function, approaches that trigger degradation of RNA could provide a general way to affect biology. Indeed, targeted RNA degradation is an effective strategy to selectively and potently modulate biology. We describe several approaches to endow small molecules with the power to cleave RNAs. Central to these strategies is Inforna, which designs small molecules targeting RNA from human genome sequence. Inforna deduces the uniqueness of a druggable pocket, enables generation of hypotheses about functionality of the pocket, and defines on- and off-targets to drive compound optimization. RNA-binding compounds are then converted into cleavers that degrade the target directly or recruit an endogenous nuclease to do so. Cleaving compounds have significantly contributed to understanding and manipulating biological functions. Yet, there is much to be learned about how to affect human RNA biology with small molecules.
- Subjects :
- 0301 basic medicine
Small interfering RNA
RNA Stability
Druggability
Drug Evaluation, Preclinical
Computational biology
010402 general chemistry
01 natural sciences
Biochemistry
Article
Analytical Chemistry
Small Molecule Libraries
03 medical and health sciences
Bleomycin
Structure-Activity Relationship
Humans
Molecular Targeted Therapy
RNA, Small Interfering
Nuclease
Genome
biology
Base Sequence
Chemistry
RNA
Non-coding RNA
Small molecule
0104 chemical sciences
030104 developmental biology
Drug Design
Mutation
Nucleic acid
biology.protein
Nucleic Acid Conformation
Function (biology)
Subjects
Details
- Language :
- English
- Database :
- OpenAIRE
- Journal :
- Curr Opin Chem Biol
- Accession number :
- edsair.doi.dedup.....220b1e760f618aea26881c4504bc01bc