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Authors
Khare, Veenita https://orcid.org/0000-0003-0677-8967
Farré, Jean-Claude
Ait Kbaich, Mouad https://orcid.org/0000-0001-7508-6935
Rocca, Céline J https://orcid.org/0000-0002-8706-3869
Tang, Cynthia
Ma, Xuan https://orcid.org/0000-0002-1040-3781
Biederman, Kavya https://orcid.org/0009-0006-0043-4492
Mathur, Ioli https://orcid.org/0009-0001-8575-8149
Badell-Grau, Rafael A https://orcid.org/0000-0001-7640-822X
Sivakumar, Anusha
Chen, Rola https://orcid.org/0009-0004-1604-0290
Catz, Sergio D
Cherqui, Stephanie https://orcid.org/0000-0003-1240-5219
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Funding
Funding for this research was provided by:
Cystinosis Research Foundation
California Institute for Regenerative Medicine (CLIN2-11478 and TRAN1-13983)
HHS | NIH | National Institute of Neurological Disorders and Stroke (R01-NS108965,R01NS135162,P30-NS047101)
HHS | NIH | National Institute on Aging (R01-AG086443)
National MPS Society
Friedreich's Ataxia Research Alliance
HHS | NIH | National Institute of Diabetes and Digestive and Kidney Diseases (R01DK110162)
HHS | NIH | National Heart, Lung, and Blood Institute (P01HL152958)
Office of Research Infrastructure Programs (NIH S10 grant S10OD030417)
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Article History
Received: 4 June 2025
Revised: 12 February 2026
Accepted: 13 February 2026
First Online: 24 March 2026
Disclosure and competing interests statement
: Stephanie Cherqui is a cofounder, shareholder, and a member of both the Scientific Board and board of directors of Papillon Therapeutics Inc. Stephanie Cherqui is also the Chair of the Scientific Review Board and a member of Board of Trustees of the Cystinosis Research Foundation. This work is covered in the patent entitled “Methods of Treating Lysosomal Disorders” (#US-2024-0009247-A1). Anusha Sivakumar is a consultant for Papillon Therapeutics, Inc and receives income. 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. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
