利用基因转移规避脱靶效应。

Using gene transfer to circumvent off-target effects.

作者信息

Miller C P, Blau C A

机构信息

Department of Medicine/Hematology, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA 98195, USA.

出版信息

Gene Ther. 2008 May;15(10):759-64. doi: 10.1038/gt.2008.43. Epub 2008 Mar 20.

DOI:10.1038/gt.2008.43

PMID:18356813

Abstract

Many recombinant growth factors have failed in clinical trials due to off-target effects. We describe a method for circumventing off-target effects that involves equipping cells with a conditionally active signaling protein that can be specifically activated by an exogenously administered synthetic ligand. We believe that this approach will have many applications in gene and cell therapy.

摘要

许多重组生长因子因脱靶效应在临床试验中失败。我们描述了一种规避脱靶效应的方法，该方法涉及为细胞配备一种可被外源性施用的合成配体特异性激活的条件活性信号蛋白。我们相信这种方法将在基因和细胞治疗中有许多应用。