Study concept and design: Hacein-Bey Abina, Gaspar, McNicol, Hara, Lefrère, Honnet, Fischer, Thrasher, Galy, Cavazzana.
Acquisition, analysis, or interpretation of data: Hacein-Bey Abina, Gaspar, Blondeau, Caccavelli, Charrier, Buckland, Picard, Six, Himoudi, Gilmour, Xu-Bayford, Rivat, Touzot, Mavilio, Lim, Tréluyer, Héritier, Lefrère, Magalon, Pengue-Koyi, Blanche, Sherman, Male, Berry, Malani, Bushman, Fischer, Thrasher, Galy, Cavazzana.
Drafting of the manuscript: Hacein-Bey Abina, Gaspar, Bushman, Thrasher, Galy.
Critical revision of the manuscript for important intellectual content: Hacein-Bey Abina, Blondeau, Caccavelli, Charrier, Buckland, Picard, Six, Himoudi, Gilmour, McNicol, Hara, Xu-Bayford, Rivat, Touzot, Mavilio, Lim, Tréluyer, Héritier, Lefrère, Magalon, Pengue-Koyi, Honnet, Blanche, Sherman, Male, Berry, Malani, Fischer, Galy, Cavazzana.
Statistical analysis: Sherman, Berry, Malani, Bushman, Thrasher.
Obtained funding: Gaspar, Fischer, Thrasher, Galy, Cavazzana.
Administrative, technical, or material support: Hacein-Bey Abina, Gaspar, Blondeau, Caccavelli, Charrier, Buckland, Picard, Six, Himoudi, Gilmour, Hara, Xu-Bayford, Touzot, Lim, Tréluyer, Héritier, Magalon, Pengue-Koyi, Honnet, Blanche, Sherman, Bushman, Fischer, Thrasher, Galy, Cavazzana.
Study supervision: Hacein-Bey Abina, Gaspar, Gilmour, Mavilio, Héritier, Magalon, Fischer, Thrasher, Galy, Cavazzana.
Conflict of Interest Disclosures: All authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Buckland reports grants from Genethon, Great Ormond Street Hospital, the European Commission, the Medical Research Council, and the Wellcome Trust. Dr McNicol reports grants from the National Institute for Health Research Imperial Biomedical Research Centre. Dr Touzot reports grants from the European Research Council, CELL-PID, and Genethon. Dr Lefrère reports board membership for Teva, payment for lectures/speakers bureaus from Amgen, and payment for development of educational presentations from Bristol-Myers Squibb. Dr Berry reports grants from the National Institutes of Health. No other disclosures were reported.
Funding/Support: This study was sponsored by Genethon, Evry, France.
Role of the Funder/Sponsor: The sponsor, as represented by Drs Charrier, Mavilio, Honnet, and Galy, was involved in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, and approval of the manuscript; and decision to submit the manuscript for publication.
Additional Contributions: We are grateful for expert help from Didier Caizergues, DPharm, Malika Souquières, MS, Estelle de Barbeyrac, PhD, Marie-Laurence Gourlay-Chu, MD, and Hafedh Haddad, MD, who are employees of Genethon and helped in preparation and filing of regulatory approvals and clinical study management. We acknowledge Shabi Soheili, PhD, and Alessandra Magnani, PhD, Necker Children’s Hospital, for their expert assistance in the characterization of the natural killer and B-cell population. We thank Valérie Jolaine, MS, and Elodie Henry, MS, Assistance Publique–Hôpitaux de Paris at the URC-CIC Paris Center, for implementation, monitoring, and data management of the study. We thank Guillaume Corre, PhD, postdoctoral fellow at Genethon, for useful discussions on data analysis. We are also grateful to the Genethon bioproduction and control and quality assurance teams for providing the clinical-grade vector for the studies. We thank the patients’ families for their continuous support of the study, as well as the medical and nursing staff of the Immunology and Pediatric Hematology Department, Necker Children’s Hospital, Paris, and the Immunology and Bone Marrow Transplantation staff at Great Ormond Street Hospital NHS Trust, London. We also acknowledge expert scientific and medical input from William Vainchenker, MD, PhD, INSERM U1009, Institut Gustave Roussy, Villejuif, France. No financial compensation was received.