Clinical Development of Gene Therapies: The First Three Decades and Counting.
Clinical Development of Gene Therapies: The First Three Decades and Counting.
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DOI：
10.1016/j.omtm.2020.10.004
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发表时间：
2020-12-11
期刊：
影响因子：
--
通讯作者：
Puri RK
中科院分区：
文献类型：
--
作者：
Lapteva L;Purohit-Sheth T;Serabian M;Puri RK
在过去的三十年中，基因治疗领域取得了显着的进步，从单纯的实验室实验激增到食品和药物管理局（FDA）批准的产品，这些产品为以前没有治疗选择的严重疾病患者带来了疾病负担的显着减少。在本文中，我们回顾了基因治疗临床研究的发展，并描述了FDA在1988-2019年收到的研究性新药申请中评估的基因治疗产品开发计划。我们还讨论了在美国批准的前六个溶瘤和基因治疗产品的临床开发计划。通讯作者及其同事回顾了基因治疗领域的临床研究，并反思了自第一次基因治疗临床试验以来该领域的趋势和发展。他们还强调了前六个FDA批准的溶瘤和基因治疗产品的特点。
In the past three decades the field of gene therapy has made remarkable progress, surging from mere laboratory experiments to Food and Drug Administration (FDA)-approved products that bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. Herein, we review the evolution of the gene therapy clinical research landscape and describe the gene therapy product development programs evaluated by the FDA in Investigational New Drug applications received in 1988–2019. We also discuss the clinical development programs of the first six oncolytic and gene therapy products approved in the United States. The corresponding author and colleagues review clinical research in the field of gene therapies and reflect on the trends and developments occurring in the field since the first gene therapy clinical trial. They also highlight features of the first six FDA-approved oncolytic and gene therapy products.
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影响因子：
158.5
作者：
ROSENBERG, SA;AEBERSOLD, P;ANDERSON, WF
通讯作者：
ANDERSON, WF
影响因子：
--
作者：
Lee, KC
通讯作者：
Lee, KC
影响因子：
4
作者：
Chung, Daniel C.;McCague, Sarah;High, Katherine A.
通讯作者：
High, Katherine A.
影响因子：
4.2
作者：
Maier, Dawn A.;Brennan, Andrea L.;Levine, Bruce L.
通讯作者：
Levine, Bruce L.
影响因子：
3.3
作者：
Al-Zaidy, Samiah A.;Kolb, Stephen J.;Mendell, Jerry R.
通讯作者：
Mendell, Jerry R.