Tamura, Serena
Nelson, Andrew D.
Spratt, Perry W. E.
Hamada, Elizabeth C. https://orcid.org/0009-0008-0614-8141
Zhou, Xujia
Kyoung, Henry
Li, Zizheng
Arnould, Coline
Barskyi, Vladyslav
Krupkin, Beniamin https://orcid.org/0000-0002-7457-7342
Young, Kiana
Zhao, Jingjing
Holden, Stephanie S.
Sahagun, Atehsa https://orcid.org/0000-0001-7521-3279
Keeshen, Caroline M.
Lu, Congyi
Ben-Shalom, Roy
Taloma, Sunrae E. https://orcid.org/0000-0002-7210-8614
Schamiloglu, Selin
Li, Ying C. https://orcid.org/0009-0009-4792-8336
Min, Lia https://orcid.org/0000-0002-3977-1684
Jenkins, Paul M. https://orcid.org/0000-0002-4207-5823
Pan, Jen Q.
Paz, Jeanne T. https://orcid.org/0000-0001-6339-8130
Sanders, Stephan J. https://orcid.org/0000-0001-9112-5148
Matharu, Navneet
Ahituv, Nadav https://orcid.org/0000-0002-7434-8144
Bender, Kevin J. https://orcid.org/0000-0001-7084-1532
Article History
Received: 23 January 2024
Accepted: 13 August 2025
First Online: 17 September 2025
Change Date: 23 February 2026
Change Type: Update
Change Details: In the version of the article initially published, in the “CRISPRa in vitro optimization” section of the Methods, the text “rAAV vectors were generated using similar plasmids and cloning methods as described previously” originally cited ref. 1 but has now been corrected to ref. 17 in the HTML and PDF versions of the article.
Change Date: 8 December 2025
Change Type: Correction
Change Details: A Correction to this paper has been published:
Change Details: https://doi.org/10.1038/s41586-025-09995-9
Competing interests
: N.M. is the cofounder and former board member and CSO of Regel Therapeutics, N.A. is the cofounder of Regel Therapeutics and both N.A. and K.J.B. are on the scientific advisory board of Regel Therapeutics. P.W.E.S. is a Program Director at Regel Therapeutics. N.M. and N.A. are the inventors on patent ‘Gene therapy for haploinsufficiency’ WO2018148256A9. N.A., K.J.B. and S.J.S. receive funding from BioMarin Pharmaceutical Incorporated. The other authors declare no competing interests.