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US 8,007,780 B2Granted
AAV vectors for gene delivery to the lung
AssigneeGenzyme Corporation
InventorsAlejandra E. Arbetman, Michael A. Lochrie
Life of this patent
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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.
Claims7
A method of providing a protein to the lung of a vertebrate subject, said method comprising:
- (a) providing an infectious recombinant AAV virion which comprises an AAV vector comprising a heterologous nucleic acid molecule encoding the protein operably linked to control elements capable of directing the in vivo expression of said 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;
- and
- (b) administering said recombinant AAV virion to said vertebrate subject, wherein said administration results in delivery of the virion to the lung and said protein is expressed in the lung.
Dependencies are inferred from the claim text.