Conditioning methods for gene therapy
OwnerAnthony BOITANO, Michael Cooke +2inventors
InventorsAnthony BOITANO, Michael Cooke, Rahul Palchaudhuri, Bradley R. Pearse
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OwnerAnthony BOITANO, Michael Cooke +2inventors
InventorsAnthony BOITANO, Michael Cooke, Rahul Palchaudhuri, Bradley R. Pearse
Estimating the term…
The disclosure provides compositions and methods useful for the depletion of a specific population of endogenous hematopoietic stem cells and/or immune cells from a subject prior to transplantation with genetically modified stem cells to improve the engraftment of the transplanted stem cells and provide gene therapy. The disclosure provides compositions and methods for the treatment of various hematopoietic diseases, metabolic disorders, cancers, and autoimmune diseases, among others. Described herein are antibodies, antigen-binding fragments, and conjugates thereof that can be applied to effect the treatment of these conditions, for instance, by depleting a population of CD117+ or CD45+ cells in a patient, such as a human.
A method of administering genetically modified stem cells to a human subject in need thereof, the method comprising:
A method of treating a human subject with genetically modified cells, the method comprising administering a transplant comprising a population of genetically modified stem cells to the human subject in need thereof, wherein the human subject has received a conditioning treatment comprising an antibody-drug conjugate (ADC) that binds to a cell surface molecule expressed on hematopoietic stem cells (HSC) and/or immune cells.
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Dependencies are inferred from the claim text.