Pharmacologically regulated gene expression in the retina following transduction with viral vectors

被引：29

作者：

Dejneka, NS

Auricchio, A

Maguire, AM

Ye, X

Gao, GP

Wilson, JM

Bennett, J

机构：

[1] Univ Penn, Scheie Eye Inst, Stellar Chance Labs 310, Inst Human Gene Therapy,Dept Ophthalmol, Philadelphia, PA 19104 USA

[2] Univ Penn, Dept Mol & Cellular Engn, Inst Human Gene Therapy, Wistar Inst, Philadelphia, PA 19104 USA

[3] Childrens Natl Med Ctr, Childrens Res Inst, Med Genet Res Ctr, Washington, DC 20010 USA

来源：

GENE THERAPY | 2001年 / 8卷 / 06期

关键词：

adenovirus; tetracycline-inducible gene expression system; enhanced green fluorescent protein; human growth hormone; retinal pigment epithelium;

D O I：

10.1038/sj.gt.3301413

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 [生物化学与分子生物学]; 081704 [应用化学];

摘要：

The availability of inducible expression systems makes regulatable control of therapeutic proteins an attainable goal in gene therapy. We delivered tetracycline-inducible transgenes to the subretinal space using recombinant adenoviruses. Upon administration of doxycycline, we demonstrated reversible expression of green fluorescent protein in the retinal pigment epithelium as well as modulation of human growth hormone produced in the retina and secreted in the blood stream. This mode of delivery and regulation offers a unique way to evaluate gene function in the eye and represents a novel method for introducing therapeutic proteins into the retina.

引用

页码：442 / 446

页数：5

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