Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.
Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA.
Blood. 2024 Mar 14;143(11):967-970. doi: 10.1182/blood.2023021598.
The root cause of sickle cell anemia has been known for 7 decades, yet no curative therapies have been available other than allogeneic bone marrow transplantation, for which applicability is limited. Two potentially curative therapies based on gene therapy and gene editing strategies have recently received US Food and Drug Administration approval. This review surveys the nature of these therapies and the opportunities and issues raised by the prospect of definitive genetically based therapies being available in clinical practice.
镰状细胞贫血的根本原因已为人所知 70 年，但除了异体骨髓移植外，尚无其他治愈疗法，而后者的适用性有限。最近，两种基于基因治疗和基因编辑策略的潜在治愈疗法获得了美国食品和药物管理局的批准。本文综述了这些疗法的性质，以及在临床实践中提供基于基因的确定性治疗方法的前景所带来的机遇和问题。
