Method of grafting genetically modified cells to treat defects, disease or damage of the central nervous system
AssigneeThe Regents of the University of California
InventorsFred H. Gage, Theodore Friedmann, Michael B. Rosenberg, Jon A. Wolff, Malcolm Schinstine, Michael D. Kawaja, Jasodhara Ray
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Abstract
Methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat defective, diseased or damaged cells are disclosed. The modified donor cells produce functional molecules that effect the recovery or improved function of cells in the CNS. Methods and vectors for carrying out gene transfer and grafting are described.
Claims74
A method for treating defective, diseased or damaged cells in the mammalian central nervous system comprising grafting donor cells into the central nervous system of a subject, said subject or donor cells treated so as to minimize graft rejection, said donor cells genetically modified by insertion of at least one transgene encoding a product or products which directly or indirectly affect the cells into said cells to produce functional molecules in a sufficient amount to ameliorate said defective, diseased or damaged cells in the central nervous system.
A method for treating defective, diseased or damaged cells in the central nervous system of a mammalian subject comprising:
- a) grafting fibroblasts from a mammal, genetically modified with a vector containing ChAT cDNA to express acetylcholine into the central nervous system of the mammal having defective, diseased or damaged cells in the central nervous system said subject or fibroblasts treated so as to minimize graft rejection;
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- b) administering choline chloride orally to maintain and enhance secretion of said acetylcholine from said fibroblasts.
A method for enhancing the expression of a therapeutic transgene product from quiescent donor cells grafted into a central nervous system of a mammalian subject, said donor cells genetically modified by insertion of a therapeutic transgene via a vector carrying said transgene, said subject or donor cells treated so as to minimize graft rejection, the method comprising inserting a promoter in said vector.
A method for treating defective, diseased or damaged cells in the central nervous system of a mammalian subject comprising grafting genetically modified primary fibroblasts into the central nervous system, said fibroblasts modified by insertion of a therapeutic transgene carried in a retrovital vector to produce the product the of said defective diseased or ed cells, said subject or fibroblasts treated so as to minimize graft rejection.
Dependencies are inferred from the claim text.