Abstract
We have evaluated the transduction profiles of an HIV-based lentiviral vector delivered regionally to ocular tissues in vivo. Following subretinal injection, a green fluorescent protein (GFP) reporter gene was efficiently and stably expressed in retinal pigment epithelial (RPE) cells. Limited transduction of adjacent photoreceptors occurred in newborn mice, but was inefficient in adult animals. Injection of the vector into the anterior chamber resulted in efficient and stable transduction of corneal endothelial cells. Efficient in vivo gene transfer into cells of the corneal endothelium and retinal pigment epithelium by lentiviral vectors may therefore offer a valuable approach to the treatment of disorders of the cornea and outer retina.
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Acknowledgements
JWBB is a Wellcome Trust Research Training Fellow. AJT is a Wellcome Trust Senior Clinical Fellow. KF and CD are supported by grants from the Primary Immunodeficiency Association and the Chronic Granulomatous Disease Research Trust.
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Bainbridge, J., Stephens, C., Parsley, K. et al. In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium. Gene Ther 8, 1665–1668 (2001). https://doi.org/10.1038/sj.gt.3301574
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DOI: https://doi.org/10.1038/sj.gt.3301574
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