Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC, USA.
Expert Rev Hematol. 2024 Sep;17(9):555-566. doi: 10.1080/17474086.2024.2386366. Epub 2024 Jul 31.
Sickle cell disease (SCD) is a monogenic disorder that exerts several detrimental health effects on those affected, ultimately resulting in significant morbidity and early mortality. There are millions of individuals globally impacted by this disease. Research in gene therapy has been growing significantly over the past decade, now with two FDA approved products, aiming to find another cure for this complex disease.
This perspective article aims to provide a clinician's insight into the current landscape of gene therapies, exploring the novel approaches, clinical advances, and potential impact on the management and prognosis of SCD. A comprehensive literature search encompassing databases such as PubMed, Web of Science and Google Scholar was employed. The search covered literature published from 1980 to 2024, focusing on SCD and curative therapy.
After careful evaluation of the risks and benefits associated with the use of gene therapy for affected patients, the need for a cure outweighs the risks associated with treatment in most cases of SCD. With advances in current technologies, gene therapies can increase access to cures for patients with SCD.
镰状细胞病（SCD）是一种单基因疾病，会对受影响者造成多种不良健康影响，最终导致高发病率和早亡。全球有数百万受这种疾病影响的人。过去十年，基因治疗的研究有了显著的增长，现在已有两种 FDA 批准的产品，旨在为这种复杂的疾病寻找另一种治疗方法。
本文旨在从临床医生的角度探讨基因治疗的现状，探索新方法、临床进展以及对 SCD 管理和预后的潜在影响。我们使用了 PubMed、Web of Science 和 Google Scholar 等数据库进行全面的文献检索。搜索涵盖了 1980 年至 2024 年发表的关于 SCD 和根治性治疗的文献。
在仔细评估了基因治疗对受影响患者的风险和益处之后，对于大多数 SCD 患者来说，治疗的益处大于风险。随着现有技术的进步，基因疗法可以增加 SCD 患者获得治疗的机会。
