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Authors
Pernaci, Carla
Johnson, Avalon
Gillette, Sydney
Warden, Anna S.
McCormick, Chad
Weiser-Novak, Sammy
Ramirez, Gabriela
Broersma, Emily H. https://orcid.org/0000-0001-8540-2807
Mishra, Priyanka
Sivakumar, Anusha https://orcid.org/0000-0002-1366-9681
Cherqui, Stephanie
Coufal, Nicole G. https://orcid.org/0000-0001-6547-1733
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Funding
Funding for this research was provided by:
California Institute for Regenerative Medicine (TRAN1-13983)
California Institute for Regenerative Medicine (EDUC2-08388)
California Institute for Regenerative Medicine (TRAN1-13983)
National Institutes of Health grant
Friedreich's Ataxia Research Alliance
Foundation for the National Institutes of Health (R01NS135162)
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Article History
Received: 31 January 2025
Accepted: 13 November 2025
First Online: 29 November 2025
Competing interests
: S.C. is a cofounder, shareholder, and member of both the Scientific Board and board of directors of Papillon Therapeutics Inc. S.C. also serves as a member of the Scientific Review Board and Board of Trustees of the Cystinosis Research Foundation. This work is covered in the patent entitled “Methods for treating Mitochondrial Disorders” #114198-3029; (inventor: S.C.; granted U.S. Patent Nos. 12,012,436, 12,012,437 and 12,011,488, issued on June 18, 2024, covering the use of the gene-edited HSPCs for Friedreich’s ataxia and the specific gene editing approach employed. A.S. is a paid consultant for Papillon Therapeutics Inc. The terms of this arrangement have been reviewed and approved by the University of California, San Diego in accordance with its conflict-of-interest policies. All other authors declare no conflict of interest.
