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:
Jan. 21, 2025

Filed:

Jun. 03, 2019
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, Wynnewood, PA (US);

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

Artur Cideciyan, Lafayette Hill, PA (US);

Samuel Jacobson, Penn Valley, PA (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1138 (2013.01); C12N 15/86 (2013.01); C12N 2310/121 (2013.01); C12N 2310/122 (2013.01); C12N 2310/14 (2013.01); C12N 2320/32 (2013.01); C12N 2750/14142 (2013.01); C12N 2750/14143 (2013.01);
Abstract

Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering 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 a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.


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