ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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7 citing papers in PubMed, 0 citations in OpenAlex.
- Gene therapy advances using canine and feline animal models of inherited retinal degeneration.Eye (London, England) · 2025Review
- Development of Inducible CD19-CAR T Cells with a Tet-On System for Controlled Activity and Enhanced Clinical Safety.International journal of molecular sciences · 2018Article
- AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- RANGE: Gene Transfer of Reversibly Controlled Polycistronic Genes.Molecular therapy. Nucleic acids · 2013Article
- Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Gene therapy for Leber congenital amaurosis: advances and future directions.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2012Review
- Gene supplementation therapy for recessive forms of inherited retinal dystrophies.Gene therapy · 2012Review
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Authors and funding
10 authors at 5 institutions in 2 countries.
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No grant is acknowledged in the PubMed record.
Abstract
In previous studies, we demonstrated that recombinant adeno-associated virus (rAAV)-mediated gene transfer of the doxycycline (Dox)-regulatable system allows for the regulation of erythropoietin (EPO) expression in the retina of nonhuman primates after intravenous or oral administration of Dox. In addition, it was shown that administrating different amounts of Dox resulted in a dose-response dynamic of transgene expression. Adeno-associated viral gene therapy has raised hope for the treatment of patients with Leber congenital amaurosis, caused by mutations in the retinal pigment epithelium (RPE)-specific gene RPE65. The preliminary results of three clinical trials suggest some improvement in visual function. However, further improvements might be necessary to optimize vision recovery and this means developing vectors able to generate transgene expression at physiological levels. The purpose of this study was to investigate the ability of the Dox-regulatable system to regulate retinal function in RPE65(-/-) Briard dogs. rAAV vectors expressing RPE65 under the control of either the TetOff and TetOn Dox-regulated promoters or the cytomegalovirus (CMV) constitutive promoter were generated and administered subretinally to seven RPE65-deficient dogs. We demonstrate that the induction and deinduction of retinal function, as assessed by electroretinography (ERG), can be achieved using a Dox-regulatable system, but do not lead to any recovery of vision.
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