Transplant for non-malignant disorders: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the role of alternative donors, stem cell sources and graft engineering.

Hematopoietic stem cell transplantation (HSCT) is curative for many non-malignant disorders. As HSCT and supportive care technologies improve, this life-saving treatment may be offered to more and more patients. With the development of new preparative regimens, expanded alternative donor availability, and graft manipulation techniques, there are many options when choosing the best regimen for patients. Herein the authors review transplant considerations, transplant goals, conditioning regimens, donor choice, and graft manipulation strategies for patients with non-malignant disorders undergoing HSCT.

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Copyright © 2023 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest AA served on the safety monitoring committee for Sangamo Therapeutics and has no financial interest in the development of gene therapies. AB serves as a consultant for Neovii, Sobi, Adicet Bio, and Cellevolve Bio. JJB serves as a consultant for AVROBIO, BlueRock Therapeutics, Race Oncology, Advanced Clinical, Omeros, Sanofi, Medexus Pharmaceuticals, Equillium, and Sobi. CB serves as a consultant for Zodiac Produtos Farmacêuticos, Amgen, and Novartis. SC serves as a consultant for, owns shares in, and receives royalties from ExCellThera. ORK serves as a consultant for Sobi. SP receives support for the conduct of clinical trials through Memorial Sloan Kettering Cancer Center from AlloVir, Atara Biotherapeutics, and Jasper Therapeutics, and is an inventor of intellectual property related to the development of a third-party virus-specific T-cell program, with all rights assigned to Memorial Sloan Kettering Cancer Center. DP's institution (Fiona Stanley Hospital) has received honoraria from Novartis, Gilead Sciences, Bristol Myers Squibb/Celgene, and Jazz Pharmaceuticals. AS has served as a consultant for Spotlight Therapeutics (2020), Medexus Pharmaceuticals (2021), and Vertex Pharmaceuticals (2021), and received research funding from CRISPR Therapeutics (2021–2022) and honoraria from Vindico Medical Education (2020). AS has also collaborated on research with Magenta Therapeutics (2021–Present) and served as clinical trial principal investigator for CRISPR Therapeutics (2018–Present), Vertex Pharmaceuticals (2018–Present), Novartis (2019–Present), and Magenta Therapeutics (2021–Present).