Methods in molecular biology · 2012 · 46 citations · 20 references
Subretinal injections in mice become increasingly important. Currently, the most prominent application is in gene therapy of inherited eye diseases by means of viral vector delivery to photoreceptors or the retinal pigment epithelium (RPE). Since there are no large animal models for most of these diseases, genetically modified mouse models are commonly used in preclinical proof-of-concept studies. However, because of the relatively small mouse eye, adverse effects of the subretinal delivery procedure itself may interfere with the therapeutic outcome. The protocol described here concerns a transscleral pars plana subretinal injection in small eyes, and may be used for but not limited to virus-mediated gene transfer.
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Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
James Bainbridge, Alexander J. Smith, Susie S. Barker et al. · New England Journal of Medicine · 2008 · 1.9K citations · Full text
Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa
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William J. Marks, Jill L. Ostrem, L. Verhagen et al. · The Lancet Neurology · 2008 · 581 citations
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