Standing on the shoulders of stem cell gene therapists: history, hyperbole and hope for the future.

Q1 Medicine

Human Gene Therapy Clinical Development Pub Date : 2016-08-30 DOI:10.1089/hum.2016.127

J. Gardner

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引用次数: 0

Abstract

A new type of medicine was approved in Europe at the end of May that culminated from the successful convergence of two fields of science: stem cell transplantation and gene therapy. Strimvelis, a patient-specific gene-modified stem cell medicine for ADA-SCID (a fatal immunometabolic disorder similar to the bubble-boy disease), was developed by scientists at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, then later partnered with GSK. The journey took over 25 years of dedicated work from many groups and involved a pivotal trial with 12 children and their brave families. I was fortunate to be involved on the GSK side of the TIGET alliance from 2010-2015, building upon my previous experiences in gene-modified stem cells during a post-doctoral fellowship in the mid-1990s and at Chiron, which had acquired Viagene, an early gene therapy biotech. I thought it was timely to pick out a couple of observations from the development of Strimvelis to see how these might apply not only for the future of stem cell gene therapy but also be the shoulders for the adjacent CAR-T and gene editing technologies to stand on.

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站在干细胞基因治疗师的肩膀上:历史、夸张和对未来的希望。

5月底，一种新型药物在欧洲获得批准，这是干细胞移植和基因治疗这两个科学领域成功融合的结果。Strimvelis是一种针对ADA-SCID(一种类似于泡泡男孩病的致命免疫代谢疾病)的患者特异性基因修饰干细胞药物，由米兰圣拉斐尔特勒松基因治疗研究所(TIGET)的科学家开发，后来与葛兰素史克合作。这段旅程花费了许多团体25年的努力，包括对12名儿童和他们勇敢的家庭的关键试验。我很幸运地从2010年到2015年参与了TIGET联盟的GSK一方，这是基于我之前在上世纪90年代中期的博士后研究期间在基因修饰干细胞方面的经验，以及在Chiron的经验。Chiron收购了早期基因治疗生物技术公司Viagene。我认为现在是时候从Strimvelis的发展中挑选出一些观察结果，看看这些观察结果如何不仅适用于干细胞基因治疗的未来，而且也适用于相邻的CAR-T和基因编辑技术。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Human Gene Therapy Clinical Development CRITICAL CARE MEDICINEMEDICINE, RESEARCH &-MEDICINE, RESEARCH & EXPERIMENTAL

CiteScore

7.20

自引率

0.00%

发文量

期刊介绍： Human Gene Therapy (HGT) is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes important advances in DNA, RNA, cell and immune therapies, validating the latest advances in research and new technologies.