The new drug not only is a boon for patients with spinal muscular atrophy, but also may spearhead the development of further chemical RNA modulators for a broad range of conditions.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Modification of BCLX pre-mRNA splicing has antitumor efficacy alone or in combination with radiotherapy in human glioblastoma cells
Cell Death & Disease Open Access 21 February 2024
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Change history
05 February 2021
A Correction to this paper has been published: https://doi.org/10.1038/s41587-021-00841-7
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Sheridan, C. First small-molecule drug targeting RNA gains momentum. Nat Biotechnol 39, 6–8 (2021). https://doi.org/10.1038/s41587-020-00788-1
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41587-020-00788-1
This article is cited by
-
Modification of BCLX pre-mRNA splicing has antitumor efficacy alone or in combination with radiotherapy in human glioblastoma cells
Cell Death & Disease (2024)
-
RNA splicing dysregulation and the hallmarks of cancer
Nature Reviews Cancer (2023)
-
Amplifying gene expression with RNA-targeted therapeutics
Nature Reviews Drug Discovery (2023)
-
RNA as an off-target for FDA-approved drugs
Nature Chemistry (2023)
-
Expression and functions of long non-coding RNA NEAT1 and isoforms in breast cancer
British Journal of Cancer (2022)