Sauer Aisha V, Di Lorenzo Biagio, Carriglio Nicola, Aiuti Alessandro
aSan Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Milan bDepartment of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
Curr Opin Allergy Clin Immunol. 2014 Dec;14(6):527-34. doi: 10.1097/ACI.0000000000000114.
This review gives an overview over the most recent progress in the field of lentiviral gene therapy for primary immunodeficiencies (PIDs). The history and state-of-the-art of lentiviral vector development are summarized and the recent advancements for a number of selected diseases are reviewed in detail. Past retroviral vector trials for these diseases, the most recent improvements of lentiviral vector platforms and their application in preclinical development as well as ongoing clinical trials are discussed.
Main focus is on the preclinical studies and clinical trials for the treatment of Wiskott-Aldrich syndrome, chronic granulomatous disease, adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID) and X-linked severe combined immunodeficiency with lentiviral gene therapy.
Gene therapy for PIDs is an effective treatment, providing potential long-term clinical benefit for affected patients. Substantial progress has been made to make lentiviral gene therapy platforms available for a number of rare genetic diseases. Although many ongoing gene therapy trials are based on ex-vivo approaches with autologous hematopoietic stem cells, other approaches such as in-vivo gene therapy or gene-repair platforms might provide further advancement for certain PIDs.
本综述概述了慢病毒基因治疗原发性免疫缺陷病（PIDs）领域的最新进展。总结了慢病毒载体开发的历史和现状，并详细回顾了一些特定疾病的最新进展。讨论了过去针对这些疾病的逆转录病毒载体试验、慢病毒载体平台的最新改进及其在临床前开发中的应用以及正在进行的临床试验。
主要关注慢病毒基因治疗威斯科特-奥尔德里奇综合征、慢性肉芽肿病、腺苷脱氨酶缺乏重症联合免疫缺陷病（ADA-SCID）和X连锁重症联合免疫缺陷病的临床前研究和临床试验。
PID的基因治疗是一种有效的治疗方法，可为受影响患者提供潜在的长期临床益处。在使慢病毒基因治疗平台可用于多种罕见遗传病方面已取得了实质性进展。尽管许多正在进行的基因治疗试验基于自体造血干细胞的体外方法，但其他方法，如体内基因治疗或基因修复平台，可能会为某些PID带来进一步进展。
