A method for efficient microglia replacement
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsMarius Wernig, Yohei Shibuya
Life of this patent
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Abstract
Methods are provided for the efficient replacement of endogenous microglia with circulation-derived cells derived from the bone marrow in an individual, the method comprising hematopoietic stem cell transplantation, and upregulation of CDMC repopulation, wherein (i) endogenous hematopoietic stem cells are ablated, (ii) exogenous hematopoietic stem cells are introduced to the patient; and (iii) a glial cell conditioning agent is administered to enhance replacement of endogenous microglia with circulation-derived cells.
Claims21
A method for the efficient replacement of endogenous microglial cells in an individual with donor circulation-derived myeloid cells (CDMC), the method comprising:
- performing hematopoietic stem cell transplantation (HSCT) by (i) ablation of endogenous hematopoietic stem cells;
- and (ii) infusion of donor hematopoietic stem cells to the individual;
- and
- (iii) administering a microglial cell conditioning factor;
- wherein a stable, high level of chimerism in the brain-resident microglial cell population is achieved.
17-19. (canceled)
A method for the efficient replacement of endogenous microglial cells in an individual with a lysosomal storage disease with circulation-derived myeloid cells (CDMC) that correct the enzymatic defect of the lysosomal storage disease, the method comprising:
- performing hematopoietic stem cell transplantation (HSCT) by (i) ablation of endogenous hematopoietic stem cells;
- and (ii) infusion of donor hematopoietic stem cells to the individual that correct the enzymatic defect of the lysosomal storage disease;
- and
- (iii) administering a microglial cell conditioning factor;
- wherein a stable, high level of chimerism in the brain-resident microglial cell population that correct the enzymatic defect of the lysosomal storage disease is achieved.
The method of claim 21, wherein the lysosomal storage disease is Gaucher disease.
23-24. (canceled)
Dependencies are inferred from the claim text.