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1型糖尿病的基因治疗:是否已准备好进入临床应用?

Gene therapy for type 1 diabetes: is it ready for the clinic?

作者信息

D'Anneo Antonella, Rood Pleunie, Bottino Rita, Balamurugan A N, He Jing, Giannoukakis Nick

机构信息

Diabetes Institute, Children's Hospital of Pittsburgh, Pittsburgh 15213, USA.

出版信息

Immunol Res. 2006;36(1-3):83-9. doi: 10.1385/IR:36:1:83.

Abstract

This review, in addition to updating the growing list of type 1 diabetes- relevant gene therapies, offers an outline of short-term objectives that can readily be met to move, at least, adenoviral and adeno-associated viral-based protocols into the clinic, first as a means of facilitating islet allografts as well as platforms with which to introduce immunoregulatory transgenes. A wide array of genes have been tested to restore insulin production, to drive the differentiation of insulin-producing progenitors, and to confer immunosuppression in an antigen- and tissue-restricted manner.

摘要

本综述除了更新与1型糖尿病相关的基因治疗方法不断增加的列表外,还概述了一些短期目标,这些目标至少可以很容易地实现,从而将基于腺病毒和腺相关病毒的方案推进到临床,首先作为促进胰岛同种异体移植的一种手段,以及作为引入免疫调节转基因的平台。人们已经测试了各种各样的基因,以恢复胰岛素的产生,驱动胰岛素产生祖细胞的分化,并以抗原和组织限制的方式赋予免疫抑制作用。

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