CPC A61K 48/0058 (2013.01) [A61K 35/12 (2013.01); A61K 48/00 (2013.01); C12N 5/0621 (2013.01); C12N 5/16 (2013.01); C12N 7/00 (2013.01); C12N 15/8645 (2013.01); C12N 2510/00 (2013.01); C12N 2750/14141 (2013.01)] | 18 Claims |
1. A method of treating retinitis pigmentosa in a mammal having retinitis pigmentosa, said method comprising the intraocular administration, by injection into the subretinal space of the eye of said mammal, of mammalian primary retinal pigment epithelial (RPE) cells or induced pluripotent stem cell-derived RPE cells overexpressing mammalian orthodenticle homeobox 2 protein (OTX2 protein), thereby increasing intracellular levels of OTX2 protein in the mammal, wherein the RPE cells overexpress OTX2 protein and comprise an AAV2 viral vector comprising a nucleic acid sequence encoding an OTX2 protein comprising SEQ ID NO: 15 or SEQ ID NO: 16.
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