Journal article
Authors list: Stieger, K.; Lorenz, B.
Publication year: 2008
Pages: 1009-1023
Journal: Klinische Monatsblätter für Augenheilkunde
Volume number: 225
Issue number: 12
ISSN: 0023-2165
eISSN: 1439-3999
DOI Link: https://doi.org/10.1055/s-2008-1027607
Publisher: Georg Thieme Verlag
Abstract:
Over the last decade, significant progress has been made in the development of gene therapy strategies for the treatment of neovascular disorders and inherited dystrophies of the retina. Of all tested viral vectors, recombinant adeno-associated virus (rAAV) vectors, have been shown to be optimal vectors for gene transfer to the retina. Broadly speaking, two gene therapy strategies are used to treat retinal diseases; the first being corrective expression in the retina of the mutated gene (i.e., specific gene therapy) and the second being therapeutic expression of, for example, neurotrophic or antiangiogenic factors, in cases of neurodegenerative or neovascular, respectively, disorders (non-specific gene therapy). The naturally occurring RPE65(-/-) Briard dog model has been Successfully treated by specific gene transfer protocols and, based on these studies, the first clinical phase I trials are in preparation or have already begun. To avoid potential negative side effects due to the expression of neurotrophic and/or antiangiogenic factors in the retina, the expression of these transgenes needs to be regulated into a therapeutic window. Several regulatory systems have been tested in the retina of large animal model!; and may soon be used in clinical applications.
Citation Styles
Harvard Citation style: Stieger, K. and Lorenz, B. (2008) The Treatment of Inherited Dystrophies and Neovascular Disorders of the Retina by rAAV-Mediated Gene Therapy, Klinische Monatsblätter für Augenheilkunde, 225(12), pp. 1009-1023. https://doi.org/10.1055/s-2008-1027607
APA Citation style: Stieger, K., & Lorenz, B. (2008). The Treatment of Inherited Dystrophies and Neovascular Disorders of the Retina by rAAV-Mediated Gene Therapy. Klinische Monatsblätter für Augenheilkunde. 225(12), 1009-1023. https://doi.org/10.1055/s-2008-1027607
Keywords
AAV; ADENOASSOCIATED VIRAL VECTORS; ANIMAL-MODELS; CILIARY NEUROTROPHIC FACTOR; ENDOTHELIAL GROWTH-FACTOR; EPITHELIUM-DERIVED FACTOR; Gene therapy; hereditary disorders of the retina; LONG-TERM EVALUATION; neovascular disorders of the retina; REGULATED TRANSGENE EXPRESSION; regulatory systems; REPLACEMENT THERAPY
