Modification of sugar metabolic processes in transgenic cells, tissues and animals
AssigneeUniv. of Pittsburgh of the Commonwealth System of Higher Education, Office of Technology Management
InventorChihiro Koike
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Abstract
The present invention provides natural or transgenic galactose deficient cells, tissues, organs and animals that have been genetically modified to compensate for the abnormalities in galactose metabolic pathways. The present invention modifies sugar metabolic pathways to to prevent the deleterious accumulation of sugar metabolites in animals, tissues, organs, cells and cell lines that possess natural or transgenic abnormalities in the sugar metabolic pathways. Such cells, tissues, organs and animals can be used in research and medical therapy, including xenotransplantation.
Claims35
A galactose deficient cell comprising a genetic modification that results in expression of a protein of a galactose metabolic pathway wherein the expression of the protein reduces the accumulation of a toxic galactose metabolite in the cell.
A method to reduce the toxic accumulation of galactose metabolites in a galactose deficient cell comprising expressing a protein of a galactose metabolic pathway wherein the expression of the protein reduces the accumulation of the toxic metabolite.
A method to prepare a cell for xenotransplantation comprising:
- (a) inactivating at least one allele of a gene, wherein the gene is selected from the group consisting of alpha-1,3-galactosyltransferase, Forssman synthetase and isoGloboside 3 synthase wherein inactivation of the gene results in toxic accumulation of a galactose metabolite;
- and
- (b) expressing a protein of a galactose metabolic pathway in the cell wherein the expression of the protein reduces the accumulation of the toxic metabolite.
Dependencies are inferred from the claim text.