IP Library › Granted Patent US 7,427,396
Granted Patent B2
US 7,427,396 · App. 11/143,866 · Granted Sep 23, 2008

AAV vectors for gene delivery to the lung

Assignee: Genzyme Corporation
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Quick Facts
Patent No.
US 7,427,396
App. No.
11/143,866
Granted
Sep 23, 2008
Kind
B2
Abstract

Methods of making and using recombinant AAV vectors and virions for gene delivery to the lung are described. The recombinant AAV virions are derived from caprine AAV and bovine AAV, both of which display tropism for lung tissue.

Claims (5)

1. An infectious recombinant adeno-associated virus (AAV) virion comprising:

an AAV vector comprising a heterologous nucleic acid molecule encoding a protein, wherein the nucleic acid molecule is flanked on each end with an AAV inverted terminal repeat, wherein the AAV vector is encapsidated by a viral capsid comprising caprine VP1.

2. The virion of claim 1 , wherein the caprine VP1 comprises the amino acid sequence of SEQ ID NO: 4.

3. The virion of claim 1 , wherein the therapeutic protein is selected from the group consisting of CFTR, alpha-1-antitrypsin, superoxide dismutase (SOD), interleukin-1, interleukin-2, interleukin-3, interleukin-4, gamma-interferon, p53, and the retinoblastoma gene product.

4. The virion of claim 3 , wherein the therapeutic protein is CFTR.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 6, 2005
From: ARBETMAN, ALEJANDRA E.; LOCHRIE, MICHAEL A.
To: AVIGEN, INC
Reel/Frame 016855/0347 →
Continuity (3)
Provisional Application 6058203700 · Jun 21, 2004
Provisional Application 6057650600 · Jun 3, 2004
Related Publication 20060018841A1 · Jan 26, 2006