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L. J. Stein, D. C. Chung, J. Bennicelli, Z. Wei, J. Bennett; Gene Targeting to Murine Retinal Bipolar Cells. Invest. Ophthalmol. Vis. Sci. 2009;50(13):3010.
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Histological sections were evaluated for eGFP expression and colabeling of protein kinase C-2 (PKC-2), under fluorescence microscopy. Animals injected with AAV2/8 had extensive expression in the retinal bipolar cells, and moderate expression in retinal ganglion cells. AAV2/5 also resulted in expression in bipolar cells, but to a lesser degree than AAV2/8, but had additional expression in retinal pigment epithelial cells (RPE). AAV2/2 and 2/1 resulted in sparse expression in ganglion cells and RPE cells. Immunofluorescence revealed transduction of retinal bipolar cells in animals injected with AAV2/8 and 2/5. Delivery of the vector was well tolerated locally and systemically and animals showed no alteration in behavior or general well being.
Adeno-associated virus 2/8 exhibits strong tropism to retinal bipolar cells and efficiently delivers transgenes to retinal bipolar cells. This vector could be useful in probing the biology of retinal bipolar cells and in strategies aimed at preventing or reversing blindness.
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