The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Sep. 14, 2021

Filed:

Mar. 01, 2017
Applicants:

University of Florida Research Foundation, Incorporated, Gainesville, FL (US);

The Trustees of the University of Pennsylvania, Philadelphia, PA (US);

Inventors:

Alfred S. Lewin, Gainesville, FL (US);

William W. Hauswirth, Gainesville, FL (US);

Michael T. Massengill, Gainesville, FL (US);

William Beltran, Philadelphia, PA (US);

Gustavo D. Aguirre, Media, PA (US);

Artur Cideciyan, Lafayette Hill, PA (US);

Samuel Jacobson, Penn Valley, PA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); C12N 15/113 (2010.01); A61P 27/02 (2006.01); C12N 7/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1138 (2013.01); A61P 27/02 (2018.01); C12N 7/00 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/122 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2330/51 (2013.01); C12N 2750/14143 (2013.01);
Abstract

Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.


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