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

Gene Therapy volume 8, pages 442–446 (2001)Cite this article

Abstract

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.

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Acknowledgements

This work was supported by the Fight for Sight research division of Prevent Blindness America (PD99032; postdoctoral research fellowship) to NSD, Telethon Italia (371/B) to AA, NIHR01 EY10820 and EY12156 (JB), Foundation Fighting Blindness, NIHP30 DKY7757–05AHA (JMW), the Mackall Foundation Trust and the FM Kirby Foundation.

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Author notes

  1. N S Dejneka and A Auricchio: These two authors contributed equally to the work presented

Authors and Affiliations

  1. Department of Ophthalmology, Scheie Eye Institute, Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA, USA

    N S Dejneka, A M Maguire & J Bennett

  2. Department of Molecular and Cellular Engineering, The Wistar Institute, Institute of Human Gene Therapy, University of Pennsylvania, Philadelphia, PA, USA

    A Auricchio, G-P Gao & J M Wilson

  3. Center for Genetic Medicine Research, Children's Research Institute, Children's National Medical Center, Washington, DC, USA

    X Ye

Authors
  1. N S Dejneka
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  2. A Auricchio
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  3. A M Maguire
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  4. X Ye
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  5. G-P Gao
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  6. J M Wilson
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  7. J Bennett
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Dejneka, N., Auricchio, A., Maguire, A. et al. Pharmacologically regulated gene expression in the retina following transduction with viral vectors. Gene Ther 8, 442–446 (2001). https://doi.org/10.1038/sj.gt.3301413

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