Use of adeno-associated virus (AAV) to deliver genes
First Claim
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1. A method for transferring genetic material to primary pancreatic islet β
- -cells, comprising transducing said pancreatic islet β
-cells with a recombinant adeno associated virus vector wherein said pancreatic islet β
-cells are transduced outside of the body.
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Abstract
This invention pertains to a method of protecting pancreatic β-cells from immune system-mediated cell damage through transduction of the cells with an adeno-associated viral vector encoding cytoprotective genes. These protected cells are suitable for transplantation into mammals and will be protected from rejection by the host. The method also may be used to protect pancreatic islet β-cells in situ for the primary prevention of immune-mediated destruction which leads to Type 1 diabetes. Pancreatic β-cells may be transduced with AAV vectors according to the invention for in vivo or ex vivo gene therapy.
20 Citations
18 Claims
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1. A method for transferring genetic material to primary pancreatic islet β
- -cells, comprising transducing said pancreatic islet β
-cells with a recombinant adeno associated virus vector wherein said pancreatic islet β
-cells are transduced outside of the body. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9)
- -cells, comprising transducing said pancreatic islet β
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10. Isolated primary pancreatic islet β
- -cells harboring a recombinant adeno associated virus vector having inserted therein genetic material wherein said genetic material encodes a peptide sequence selected from the group consisting of manganese superoxide dismutase, thioredoxin, interleukin-12 antagonist p40(2), glutathione peroxidase, catalase, 15-lipoxygenase, interleukin-10, leptin and interleukin-4.
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11. A method of transferring genetic material to primary pancreatic islet β
- -cells, comprising;
(a) providing primary pancreatic islet β
-cells outside of the body; and
(b) transducing said primary pancreatic islet β
-cells with a recombinant adeno associated virus vector having inserted therein genetic material wherein said genetic material encodes a peptide sequence selected from the group consisting of manganese superoxide dismutase, thioredoxin, interleukin-12 antagonist p40(2), glutathione peroxidase, catalase, 15-lipoxygenase, interleukin-10, leptin and interleukin-4.- View Dependent Claims (12, 13, 14, 15, 16, 17, 18)
- -cells, comprising;
Specification
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Current AssigneeCity of Hope
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Original AssigneeCity of Hope
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InventorsPrasad, Konkal-Matt R., Nadler, Jerry L., Bleich, David
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Primary Examiner(s)McGarry, Sean
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Assistant Examiner(s)ZARA, JANE J
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Application NumberUS09/559,915Time in Patent Office942 DaysField of Search435/6, 435/69.1, 435/70.1, 435/91.1, 435/455, 435/456, 435/366, 435/375, 435/320.1, 536/23.1US Class Current435/455CPC Class CodesA61K 38/44 Oxidoreductases (1)A61K 48/00 Medicinal preparations cont...A61P 37/06 Immunosuppressants, e.g. dr...C12N 15/86 Viral vectorsC12N 2750/14143 viral genome or elements th...
