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.
Patent No.:
Date of Patent:
Apr. 27, 2021
Filed:
Nov. 18, 2016
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Lloyd G. Mitchell, Bethesda, MD (US);
Jean Bennett, Bryn Mawr, PA (US);
Jeannette Bennicelli, Philadelphia, PA (US);
Scott J. Dooley, Philadelphia, PA (US);
Lloyd G. Mitchell, Bethesda, MD (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Abstract
A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.