AI Article Synopsis
- Selectively targeting mRNA with small molecules was once thought to be a significant scientific hurdle, but recent discoveries have changed that perspective.
- The development of small molecule splicing modifiers and the drug risdiplam for spinal muscular atrophy (SMA) has proven that this method can effectively treat diseases.
- Numerous strategies are now available to target mRNA using small molecules, backed by advanced biophysical techniques and screening methods, paving the way for new, impactful medications.
Article Abstract
Not too long ago, the concept of selectively targeting mRNA with small molecules was perceived as a formidable scientific challenge. The discovery of small molecule splicing modifiers and the development of risdiplam for the treatment of spinal muscular atrophy (SMA) have firmly established proof of concept for this exciting new platform and transformed a scientific curiosity into a viable technology to target disease. Today, several approaches to target mRNA with small molecules, supported by biophysical and screening methods, are in place to deliver new drugs with high therapeutic relevance.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8201486 | PMC |
| http://dx.doi.org/10.1021/acsmedchemlett.0c00659 | DOI Listing |
Publication Analysis
Top Keywords
small molecule
8
molecule splicing
8
mrna small
8
small molecules
8
risdiplam approved
4
small
4
approved small
4
splicing modifier
4
modifier drug
4
drug blueprint
4
Similar Publications
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!