专利名称：METHOD OF TREATING OR RETARDING THE
DEVELOPMENT OF BLINDNESS
发明人：Gregory M. Acland,Gustavo D. Aguirre,Jean
Bennett,William W. Hauswirth,Samuel G.Jacobson,Albert M. Maguire
申请号：US12832282申请日：20100708
公开号：US20100272688A1公开日：20101028
摘要：A method for treating an ocular disorder characterized by the defect orabsence of a normal gene in the ocular cells of a human or animal subject involvesadministering to the subject by subretinal injection an effective amount of a recombinantadeno-associated virus carrying a nucleic acid sequence encoding the normal gene underthe control of a promoter sequence which expresses the product of the gene in theocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and thegene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can expressthe gene product in the RPE cells. Compositions for subretinal administration are usefulin this method.
申请人：Gregory M. Acland,Gustavo D. Aguirre,Jean Bennett,William W.Hauswirth,Samuel G. Jacobson,Albert M. Maguire
地址：Kennett Square PA US,Ithaca NY US,Bryn Mawr PA US,Gainesville FL US,PennValley PA US,Bryn Mawr PA US
国籍：US,US,US,US,US,US
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