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Authors
Spellman, Rebecca G.
Ha, Leillani L. https://orcid.org/0000-0003-4478-660X
Da Silva Duarte Lepez, Salomé https://orcid.org/0000-0001-9283-6777
Arruda, Elizabeth A.
Rodrigues, Emma
Swoboda, Kathryn J.
Alves, Christiano R. R. https://orcid.org/0000-0002-2646-9689
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Funding
Funding for this research was provided by:
Massachusetts General Hospital (MGH ECOR)
U.S. Department of Health & Human Services | NIH | Eunice Kennedy Shriver National Institute of Child Health and Human Development (R01HD054599)
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (R21NS108015)
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Article History
Received: 11 October 2024
Revised: 10 March 2025
Accepted: 20 March 2025
First Online: 1 April 2025
Competing interests
: CRRA and KJS are inventors on a patent filed by Mass General Brigham that describes genome engineering technologies to treat SMA. KJS was a recipient of a grant from Biogen and received clinical trial funding from AveXis and Biogen. CRRA is a consultant for Biogen and holds stocks in publicly traded companies developing gene therapies. RGS is a current employee and holds stock/stock options in Voyager therapeutics. The other authors declare no competing interests.
: Ethical approval and written informed parental consent were obtained from all participants under Institutional Ethics Review Boards at the Massachusetts General Hospital (protocol 2016P000469).
