IP Library › Granted Patent US 8,137,962
Granted Patent B2
US 8,137,962 · App. 11/713,291 · Granted Mar 20, 2012

Compositions for treating cystic fibrosis

Assignees: University of Florida Research Foundation; The Johns Hopkins University
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Quick Facts
Patent No.
US 8,137,962
App. No.
11/713,291
Granted
Mar 20, 2012
Kind
B2
Abstract

The invention relates to compositions and methods of treating cystic fibrosis. More specifically, this invention relates to the use the AAV vectors and constructs to provide gene therapy to cystic fibrosis patients.

Claims (4)

1. A recombinant adenoviral associated virus (AAV) gene therapy particle comprising a nucleic acid encoding a truncated cystic fibrosis transmembrane conductance regulator (CFTR) protein consisting of the amino acid sequence of SEQ ID NO 9, operatively linked to a promoter, and a first and a second AAV inverted terminal repeat (ITR) sequence flanking the sequence encoding the truncated CFTR protein.

2. The rAAV gene therapy particle of claim 1 , wherein the promoter is a chicken beta actin (CB) promoter or a cytomegalovirus enhancer/chicken beta-actin promoter.

3. The rAAV gene therapy particle of claim 1 , wherein the ITR nucleotide sequences are derived from AAV serotype 2 (AAV-2).

4. A pharmaceutical composition comprising the AAV gene therapy particle of claim 1 in a biocompatible pharmaceutical carrier.

Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 18, 2012
From: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION
To: THE JOHNS HOPKINS UNIVERSITY
Reel/Frame 028067/0349 →
CONFIRMATORY LICENSE Recorded Aug 21, 2008
From: UNIVERSITY OF FLORIDA
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 021426/0174 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 11, 2007
From: FLOTTE, TERENCE R.; GUGGINO, WILLIAM B.; CEBOTARU, LIUDMILA; MULLER, CHRISTIAN; SIRNINGER, JEFFREY R.
To: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
Reel/Frame 019809/0928 →
Continuity (3)
Continuation In Part PCTUS2005031576 · Sep 2, 2005
Provisional Application 60607324 · Sep 3, 2004
Related Publication 20080003204A1 · Jan 3, 2008