Individuals with transfusion-dependent beta thalassemia require a high burden of care and experience significant morbidity from the underlying disease and its treatment, which negatively impact the quality of life. Allogeneic hematopoietic stem cell transplantation offers the chance for a cure, but donor availability and transplant-related risks, especially in older patients, limit its use. Gene addition utilizing autologous CD34⁺ cells is an alternative, potentially curative, treatment option. Several clinical trials have investigated the use of lentiviral vectors containing a functional beta globin gene, including Lentiglobin BB305, GLOBE, and TNS9.3.55. The efficacy and safety data from these ongoing trials are discussed in this review.

中文翻译：

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β地中海贫血基因添加


患有输血依赖性β地中海贫血的个体需要承担很高的护理负担，并且基础疾病及其治疗会导致显着的发病率，这对生活质量产生负面影响。同种异体造血干细胞移植提供了治愈的机会，但供体可用性和移植相关风险（尤其是老年患者）限制了其使用。利用自体 CD34 ⁺ 细胞进行基因添加是一种替代的、具有潜在治愈性的治疗选择。多项临床试验研究了含有功能性 β 珠蛋白基因的慢病毒载体的使用，包括 Lentiglobin BB305、GLOBE 和 TNS9.3.55。本综述讨论了这些正在进行的试验的有效性和安全性数据。