发明名称 |
Method of Treating or Retarding the Development of Blindness |
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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申请公布号 |
US2009074723(A1) |
申请公布日期 |
2009.03.19 |
申请号 |
US20080253955 |
申请日期 |
2008.10.18 |
申请人 |
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA;THE UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.;CORNELL RESEARCH FOUNDATION, INC. |
发明人 |
ACLAND GREGORY M.;AGUIRRE GUSTAVO D.;BENNETT JEAN;HAUSWIRTH WILLIAM W.;JACOBSON SAMUEL G.;MAGUIRE ALBERT M. |
分类号 |
A61K48/00;A61K31/70;A61K31/7088;A61K35/12;A61K35/76;A61K38/45;A61P27/02;C07K14/705;C12N15/864 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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