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, Malcolm Schinstine, Jasodhara Ray, Theodore Friedmann, Michael D. Kawaja, Michael B. Rosenberg, Jon A. Wolff
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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.
Claims51
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 or reduce 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, the transgene being selected from the group consisting of genes coding for tryptophan hydroxylase, GABA-decarboxylase, enkephalin, dopa decarboxylase (AADC), ciliary neuronal trophic factor (CNTF), brain derived neurotrophic factor (BDNF), neurotrophin (NT)-3, NT-4, and basic fibroblast growth factor (bFGF).
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