Gregory M. Acland,Gustavo D. Aguirre,Jean Bennett,William W. Hauswirth,Samuel G. Jacobson,Albert M. Maguire
申请号：
US13406666
公开号：
US20120225930A1
申请日:
2012.02.28
申请国别(地区):
US
年份:
2012
代理人:
摘要：
A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.
