Multi-transgenic pigs for diabetes treatment
AssigneeRevivicor, Inc.
InventorDavid Ayares
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Abstract
The present invention provides certain animals, and in particular porcine animals, tissue and cells derived from these, which lack any expression of functional alpha 1,3 galactosyltransferase (αGT) and express one or more additional transgenes which make them suitable donors for pancreatic islet xenotransplantation. Methods of treatment and prevention of diabetes using cells derived from such animals are also provided.
Claims28
A genetically modified transgenic porcine animal, wherein the genetic modifications comprise modifications to the genome of the porcine that result in (i) the lack of any expression of functional alpha 1,3 galactosyltransferase (GTKO), (ii) incorporation of a human CD46 transgene under the control of the human CD46 promoter and expression of the human CD46 transgene in at least pancreatic islet cells and tissues;
- (iii) incorporation and expression of at least one immunosuppressant transgene under the control of an islet-specific promoter in the genome of pancreatic islet cells and tissues;
- (iv) incorporation and expression of at least one anticoagulant transgene under the control of an islet-specific promoter in the genome of pancreatic islet cells and tissues;
- (v) wherein the phenotype of the porcine is characterized by specific expression of the anticoagulant and immunosuppressant transgenes in pancreatic islet cells and tissues;
- and (vi) wherein transgenic porcine islet cells isolated from the porcine animal produce insulin and reduce the instant blood mediated inflammatory reaction (IBMIR), in comparison to non-transgenic porcine islet cells, after transplantation into a host.
Dependencies are inferred from the claim text.