Invention Grant
- Patent Title: Method of treating or retarding the development of blindness
- Patent Title (中): 治疗或延缓失明发展的方法
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Application No.: US14310015Application Date: 2014-06-20
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Publication No.: US09433688B2Publication Date: 2016-09-06
- Inventor: Gregory M. Acland , Gustavo D. Aguirre , Jean Bennett , William W. Hauswirth , Samuel G. Jacobson , Albert M. Maguire
- Applicant: The Trustees of the University of Pennsylvania , University of Florida Research Foundation, Incorporated , Cornell Research Foundation, Inc.
- Applicant Address: US PA Philadelphia US FL Gainesville US NY Ithaca
- Assignee: The Trustees of the University of Pennsylvania,University of Florida Research Foundation, Incorporated,Cornell Research Foundation, Inc.
- Current Assignee: The Trustees of the University of Pennsylvania,University of Florida Research Foundation, Incorporated,Cornell Research Foundation, Inc.
- Current Assignee Address: US PA Philadelphia US FL Gainesville US NY Ithaca
- Agency: Howson & Howson LLP
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N15/12 ; C12N15/861 ; A61K31/70 ; C07K14/705 ; C12N15/86 ; A61K38/51 ; A61K38/52 ; C12N15/864 ; C12N7/00 ; A61K38/00

Abstract:
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.
Public/Granted literature
- US20140377224A1 Method of Treating or Retarding the Development of Blindness Public/Granted day:2014-12-25
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