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Authors
Arnold, Paula Y. https://orcid.org/0000-0002-9493-7982
Selukar, Subodh https://orcid.org/0000-0002-3802-488X
Luo, Guangjin
Li, Ying
Epperly, Rebecca https://orcid.org/0000-0002-8998-0732
Gottschalk, Stephen https://orcid.org/0000-0003-3991-7468
Madden, Renee M.
Qudeimat, Amr
Sharma, Akshay https://orcid.org/0000-0003-3281-2081
Suliman, Ali Y. https://orcid.org/0000-0003-0941-2215
Talleur, Aimee C. https://orcid.org/0000-0002-6558-6706
Triplett, Brandon M. https://orcid.org/0000-0001-8220-9980
Naik, Swati https://orcid.org/0000-0001-9979-7360
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Funding
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute (1U01HL163983)
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Article History
Received: 7 February 2025
Revised: 25 April 2025
Accepted: 1 May 2025
First Online: 11 May 2025
Competing interests
: AS has received consultant fees from Spotlight Therapeutics, Medexus Inc, Vertex Pharmaceuticals, Sangamo Therapeutics, and Editas Medicine; is a medical monitor for a Conditioning SCID Infants Diagnosed Early clinical trial for which he receives financial compensation; has received research funding from CRISPR Therapeutics and honoraria from Vindico Medical Education and Blackwood CME; and is the St. Jude Children’s Research Hospital site principal investigator of clinical trials for genome editing of sickle cell disease sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics (NCT03745287), Novartis Pharmaceuticals (NCT04443907), and Beam Therapeutics (NCT05456880). The industry sponsors provide funding for the clinical trial, which includes salary support paid to the institution. AS has no direct financial interest in these therapies. SG is a member of the Data Safety Monitoring Board (DSMB) of Immatics, serves on the Scientific Advisory Board of Be Biopharma, served as a consultant for CARGO Therapeutics within the last 12 months, and has patents and patent applications in the fields of T cell and/or gene therapy for cancer. These conflicts are managed through the compliance office at St. Jude Children’s Research Hospital in accordance with their conflict-of-interest policy. All other authors have nothing to disclose.
: All methods were performed in accordance with the relevant guidelines and regulations. This retrospective study was approved by the St. Jude Children’s Research Hospital Institutional Review Board (No. 20-0635).
