GENE THERAPY FOR OCULAR DISORDERS

Title:

GENE THERAPY FOR OCULAR DISORDERS

Document Type and Number:

WIPO Patent Application WO/2018/160849

Kind Code:

A8

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.

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Inventors:

BENNETT JEAN (US)
BENNICELLI JEANNETTE (US)
SUN JUNWEI (US)
SONG JI-YUN (US)
NIKONOV SERGEI (US)

Application Number:

PCT/US2018/020470

Publication Date:

October 03, 2019

Filing Date:

March 01, 2018

Export Citation:

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Assignee:

UNIV PENNSYLVANIA (US)

International Classes:

C12N15/86; A61K35/76; A61K48/00; A61P27/02; C12N15/861

Attorney, Agent or Firm:

SCHALLER, Colleen, M. et al. (US)

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