当前位置: X-MOL 学术Blood › 论文详情
Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)
Gene therapy: WAS (not) just for kids
Blood ( IF 20.3 ) Pub Date : 2017-09-14 , DOI: 10.1182/blood-2017-08-798496
Donald B. Kohn 1
Affiliation  

In this issue of Blood , Morris et al describe application of autologous transplant/gene therapy for Wiskott-Aldrich syndrome (WAS), first demonstrated to be efficacious and safe in children, to bring relief to a severely affected adult. 1 In recent years, gene therapy for blood cell diseases in which autologous hematopoietic stem cells (HSCs) are corrected using a lentiviral vector has led to clinical benefits similar to those from allogeneic transplant. Much of the focus has been on severe diseases affecting infants and young children (severe combined immunodeficiency, X-linked adrenoleukodystrophy, metachromatic leukodystrophy) in which early intervention has the potential to prevent the development of major cumulative disease-related complications. More recently, adult patients with β-thalassemia, chronic granulomatous disease, and even X-linked severe combined immunodeficiency, previously having suboptimal responses to nonconditioned allogeneic hematopoietic stem cell transplantation, have shown good response to autologous gene therapy. 2,3 These results have now been extended to WAS.

中文翻译:

基因疗法:是(不是)只为孩子们准备的

在本期 Blood 中,Morris 等人描述了自体移植/基因疗法在 Wiskott-Aldrich 综合征 (WAS) 中的应用,首次证明在儿童中是有效且安全的,可为受严重影响的成人带来缓解。1 近年来,使用慢病毒载体纠正自体造血干细胞 (HSC) 的血细胞疾病基因治疗已带来与异基因移植相似的临床益处。大部分重点都放在影响婴儿和幼儿的严重疾病(严重联合免疫缺陷、X 连锁肾上腺脑白质营养不良、异染性脑白质营养不良)上,在这些疾病中,早期干预有可能预防与疾病相关的主要累积并发症的发展。最近,β-地中海贫血、慢性肉芽肿病、甚至 X 连锁的严重联合免疫缺陷,以前对非条件性同种异体造血干细胞移植反应欠佳,对自体基因治疗也表现出良好的反应。2,3 这些结果现已扩展到 WAS。
更新日期:2017-09-14
down
wechat
bug