同种异体移植与基因治疗：走向治愈之路。
Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
机构信息
Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 4-5140, Bethesda, MD 20892, USA; Hematology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
University of California San Francisco Benioff Children's Hospital, 747 52nd Street, Oakland CA 94609, USA.
出版信息
Hematol Oncol Clin North Am. 2022 Dec;36(6):1313-1335. doi: 10.1016/j.hoc.2022.06.007.
Abstract
Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.
摘要
镰状细胞病 (SCD) 的治疗方法包括异体人类白细胞抗原 (HLA) 匹配的同胞和半相合造血细胞移植 (HCT)、基因治疗和基因编辑。然而，目前尚无比较试验数据可以帮助选择一种治疗方法而不是另一种。需要新的策略来降低移植物排斥和移植物抗宿主病 (GVHD) 的风险，以扩大半相合 HCT。清髓性基因治疗和基因编辑也有其局限性。本文综述了过去 5 年来 SCD 治疗方法的最新研究进展。
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