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:
Jan. 31, 2023
Filed:
Mar. 01, 2018
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Jean Bennett, Bryn Mawr, PA (US);
Jeannette Bennicelli, Philadelphia, PA (US);
Junwei Sun, Philadelphia, PA (US);
Ji Yun Song, Lower Gwynedd, PA (US);
Sergei Nikonov, Swarthmore, PA (US);
The Trustees of the University of Pennsylvania, Philadelpia, PA (US);
Abstract
Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.