Mutation corrections in spinal muscular atrophy

Base editors can restore the expression of survival motor neuron protein to therapeutically beneficial levels in animal and cell models of spinal muscular atrophy. Access through your

institution Buy or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution Access Nature and 54 other Nature Portfolio

journals Get Nature+, our best-value online-access subscription $29.99 / 30 days cancel any time Learn more Subscribe to this journal Receive 12 digital issues and online access to articles

$119.00 per year only $9.92 per issue Learn more Buy this article * Purchase on SpringerLink * Instant access to full article PDF Buy now Prices may be subject to local taxes which are

calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support REFERENCES * Mailman, M. D. et al. _Genet.

Med._ 4, 20–26 (2002). Article  CAS  PubMed  Google Scholar  * Cartegni, L., Hastings, M. L., Calarco, J. A., de Stanchina, E. & Krainer, A. R. _Am. J. Hum. Genet._ 78, 63–77 (2006).

Article  CAS  PubMed  Google Scholar  * Finkel, R. S. et al. _N. Engl. J. Med._ 377, 1723–1732 (2017). Article  CAS  PubMed  Google Scholar  * Oskoui, M. et al. _J. Neurol._ 270, 2531–2546

(2023). Article  CAS  PubMed  PubMed Central  Google Scholar  * Mercuri, E. et al. _Lancet Neurol._ 20, 832–841 (2021). Article  CAS  PubMed  Google Scholar  * Alves, C. R. R. et al. _Nat.

Biomed. Eng._ https://doi.org/10.1038/s41551-023-01132-z (2023). Article  PubMed  Google Scholar  * Arbab, M. et al. _Science_ 380, eadg6518 (2023). Article  CAS  PubMed  PubMed Central 

Google Scholar  * Koblan, L. W. et al. _Nature_ 589, 608–614 (2021). Article  ADS  CAS  PubMed  PubMed Central  Google Scholar  * Levy, J. M. et al. _Nat. Biomed. Eng._ 4, 97–110 (2020).

Article  CAS  PubMed  PubMed Central  Google Scholar  * Hua, Y. et al. _Nature_ 478, 123–126 (2011). Article  ADS  CAS  PubMed  PubMed Central  Google Scholar  * Mathiesen, S. N., Lock, J.

L., Schoderboeck, L., Abraham, W. C. & Hughes, S. M. _Mol. Ther. Meth. Clin. Dev._ 19, 447–458 (2020). Article  CAS  Google Scholar  * Gonzalez-Sandoval, A. et al. _Nat. Commun._ 14,

2448 (2023). Article  ADS  CAS  PubMed  PubMed Central  Google Scholar  * Porto, E. M., Komor, A. C., Slaymaker, I. M. & Yeo, G. W. _Nat. Rev. Drug Discov._ 19, 839–859 (2020). Article 

CAS  PubMed  PubMed Central  Google Scholar  * Du, M., Jillette, N., Zhu, J. J., Li, S. & Cheng, A. W. _Nat. Commun._ 11, 2973 (2020). Article  ADS  CAS  PubMed  PubMed Central  Google

Scholar  * Katrekar, D. et al. _Nat. Biotech._ 40, 938–945 (2022). Article  CAS  Google Scholar  Download references AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Department of

Bioengineering, University of California, San Diego, La Jolla, CA, USA Andrew Portell & Prashant Mali Authors * Andrew Portell View author publications You can also search for this

author inPubMed Google Scholar * Prashant Mali View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence to Prashant Mali.

ETHICS DECLARATIONS COMPETING INTERESTS P.M. is a scientific co-founder of Navega Therapeutics, Boundless Biosciences, Shape Therapeutics and Engine Biosciences. The terms of these

arrangements have been reviewed and approved by the University of California, San Diego, in accordance with its conflict-of-interest policies. RIGHTS AND PERMISSIONS Reprints and permissions

ABOUT THIS ARTICLE CITE THIS ARTICLE Portell, A., Mali, P. Mutation corrections in spinal muscular atrophy. _Nat. Biomed. Eng_ 8, 111–113 (2024). https://doi.org/10.1038/s41551-023-01166-3

Download citation * Published: 21 December 2023 * Issue Date: February 2024 * DOI: https://doi.org/10.1038/s41551-023-01166-3 SHARE THIS ARTICLE Anyone you share the following link with will

be able to read this content: Get shareable link Sorry, a shareable link is not currently available for this article. Copy to clipboard Provided by the Springer Nature SharedIt

content-sharing initiative