Method of treating or retarding the development of blindness

US9433688B2 · kind B2 · utility

Assignees

• The Trustees of the University of Pennsylvania · US
• University of Florida Research Foundation, INC. · US
• Cornell Research Foundation, Inc. · US

Inventors

• Gregory M. Acland · Kennett Square, US
• Gustavo D. Aguirre · Philadelphia, US
• Jean Bennett · Via Enrico Fermi, US
• William W. Hauswirth · Gainesville, US
• Samuel G. Jacobson · Levittown, US
• Albert M. Maguire · Philadelphia, US

Key dates

• Filing date: Jun 20, 2014
• Grant date: Sep 6, 2016
• Expiry date: Jun 20, 2034

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2840/203
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.