发明申请
- 专利标题: Method of Treating or Retarding the Development of Blindness
- 专利标题(中): 治疗或阻碍盲症发展的方法
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申请号: US12253955申请日: 2008-10-18
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公开(公告)号: US20090074723A1公开(公告)日: 2009-03-19
- 发明人: Gregory M. Acland , Gustavo D. Aguirre , Jean Bennett , William W. Hauswirth , Samuel G. Jacobson , Albert M. Maguire
- 申请人: Gregory M. Acland , Gustavo D. Aguirre , Jean Bennett , William W. Hauswirth , Samuel G. Jacobson , Albert M. Maguire
- 申请人地址: US PA Philadelphia US FL Gainesville US NY Ithaca
- 专利权人: The Trustees of the University of Pennsylvania,The University of Florida Research Foundation, Inc.,Cornell Research Foundation, Inc.
- 当前专利权人: The Trustees of the University of Pennsylvania,The University of Florida Research Foundation, Inc.,Cornell Research Foundation, Inc.
- 当前专利权人地址: US PA Philadelphia US FL Gainesville US NY Ithaca
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; A61K31/7088 ; A61K35/12 ; A61K35/76 ; A61P27/02
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.
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