Gene therapy technology applied to disorders of glucose metabolism: promise, achievements, and prospects.

Gene therapy technology applied to disorders of glucose metabolism: promise, achievements, and prospects.
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基因治疗技术应用于葡萄糖代谢紊乱:前景、成就和前景。

DOI:
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发表时间:
2003
期刊:
影响因子:
2.7
通讯作者:
M. Trucco
M. Trucco
中科院分区:
工程技术4区
文献类型:
--
作者:
N. Giannoukakis;M. Trucco

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基因转移技术已经催生了一个完整的临床研究领域,统称为“基因治疗”。本文就基因治疗在预防和治疗糖代谢紊乱,特别是糖尿病方面的可行性和成就进行了综述。虽然大量的努力已经产生了基于腺病毒、逆转录病毒和单纯疱疹病毒DNA的基因递送载体,但成功的临床应用的数量并不令人印象深刻。尽管在体外和动物模型中取得了许多成功,但尚未尝试在人体中进行初步的安全性试验。本文概述了这一科学的现状,强调了将基因转移技术与细胞治疗相结合的必要性。将基因组合离体转移到多种细胞类型中可能证明比直接体内载体转移在治疗上更可行。目前旨在评估糖尿病基因治疗未来的努力至少必须考虑到将成功的方法转移到人体安全试验的重要性。
Gene transfer technology has spawned an entire realm of clinical investigation, collectively referred to as "gene therapy." The feasibility and achievements of gene therapy to prevent and treat glucose homeostasis disorders, with particular emphasis on diabetes mellitus, are evaluated in this review. While a considerable amount of effort has yielded gene delivery vectors based on adenoviral, retroviral, and herpes simplex virus DNA, the number of successful clinical applications has not been as impressive. Despite the number of successes in vitro and in animal models, preliminary safety trials in humans have not yet been attempted. The current state of this science, outlined here, underlines the necessity of marrying gene transfer technology with cell therapy. The ex vivo transfer of gene combinations into a variety of cell types will likely prove more therapeutically feasible than direct in vivo vector transfer. Current efforts aimed at assessing the future of gene therapy for diabetes must, at the very least, take into account the importance of moving successful methods into human safety trials.
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