ABSTRACT Gene transfer of neurotrophic or antiangiogenic factors has been shown to improve photoreceptor survival in retinal degenerative disorders (that is retinitis pigmentosa) and to

prevent neovascularization in retinal vascular diseases (that is age-related macular degeneration, diabetic retinopathy). Expression of such neurotrophic or antiangiogenic factors after gene

transfer requires the use of a regulatory system to control transgene expression to avoid unwanted side effects in cases of overexpression. In a previous study, we demonstrated that

rAAV-mediated gene transfer of the tetracycline-regulatable (tetR) system allows transgene regulation in the retina of nonhuman primates after intravenous administration of doxycycline

clinically used in humans (5 mg kg−1 per day) is capable to continuously induce transgene expression in all macaques tested for 6 months. Moreover, control of transgene expression persists

up to 4 years post-subretinal injection, with maximal induced levels of transgene product remaining stable over time. Access through your institution Buy or subscribe This is a preview of

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OF GENE THERAPY FOR _RLBP1_-ASSOCIATED RETINAL DYSTROPHY: A PHASE 1/2 TRIAL Article Open access 10 September 2024 HUMAN MINIPROMOTERS FOR OCULAR-RAAV EXPRESSION IN ON BIPOLAR, CONE, CORNEAL,

ENDOTHELIAL, MÜLLER GLIAL, AND PAX6 CELLS Article Open access 02 February 2021 LATE GENE THERAPY LIMITS THE RESTORATION OF RETINAL FUNCTION IN A MOUSE MODEL OF RETINITIS PIGMENTOSA Article

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ACKNOWLEDGEMENTS We thank Matthew Ellinwood for critical reading and editing. We also thank the Vector Core (www.vectors.nantes.inserm.fr) at the University Hospital of Nantes, supported by

the Association Française contre les Myopathies (AFM), the INSERM, and the Fondation pour la Thérapie Génique en Pays de la Loire. This work was also supported by the French Lions Club and

the Lions Clubs International Foundation (LCIF). AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * INSERM UMR U649, CHU-Hotel Dieu, Nantes, France K Stieger, A Mendes-Madeira, D Nivard, N

Provost, P Moullier & F Rolling * Etablissement Fran, A Mendes-Madeira, D Nivard & P Moullier * ç, A Mendes-Madeira, D Nivard & P Moullier * ais du Sang, Pays de la Loire,

Nantes, France A Mendes-Madeira, D Nivard & P Moullier * CHU-Hotel Dieu, Service d'Ophtalmologie, Nantes, France G L Meur & M Weber * Ecole Nationale Vétérinaire de Nantes,

Services d'Urgences, Nantes, France J-Y Deschamps * Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL, USA, P Moullier Authors * K Stieger View

author publications You can also search for this author inPubMed Google Scholar * A Mendes-Madeira View author publications You can also search for this author inPubMed Google Scholar * G L

Meur View author publications You can also search for this author inPubMed Google Scholar * M Weber View author publications You can also search for this author inPubMed Google Scholar * J-Y

Deschamps View author publications You can also search for this author inPubMed Google Scholar * D Nivard View author publications You can also search for this author inPubMed Google

Scholar * N Provost View author publications You can also search for this author inPubMed Google Scholar * P Moullier View author publications You can also search for this author inPubMed 

Google Scholar * F Rolling View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence to F Rolling. RIGHTS AND PERMISSIONS

Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Stieger, K., Mendes-Madeira, A., Meur, G. _et al._ Oral administration of doxycycline allows tight control of transgene

expression: a key step towards gene therapy of retinal diseases. _Gene Ther_ 14, 1668–1673 (2007). https://doi.org/10.1038/sj.gt.3303034 Download citation * Received: 26 July 2007 * Revised:

30 August 2007 * Accepted: 30 August 2007 * Published: 04 October 2007 * Issue Date: December 2007 * DOI: https://doi.org/10.1038/sj.gt.3303034 SHARE THIS ARTICLE Anyone you share the

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Nature SharedIt content-sharing initiative KEYWORDS * Doxycycline-regulated transgene expression * retina * nonhuman primate * AAV vectors * Epo * oral administration