Genetically modified hspcs resistant to ablation regime
AssigneeForty Seven, Inc.
InventorsCraig Gibbs, Jens-Peter Volkmer, Irving L. Weissman
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Abstract
The invention provides genetically modified hematopoietic stem or progenitor cells (HSPCs) and methods of using the HSPCs in stem cell replacement therapy. The HSPCs are genetically modified to express a receptor conferring a selective advantage on the introduced cells relative to endogenous HSPCs or a control HSPCs without the modification. The presence of such a receptor provides resistance to an immunotherapy regime used for eliminating endogenous HSPCs.
Claims37
A hematopoietic stem or progenitor cell (HSPC) genetically modified to express a receptor conferring a selective proliferation advantage on the genetically modified HSPC on introduction into a subject relative to endogenous HSPCs.
A method of modifying an HSPC comprising introducing into the HSPC a construct that is incorporated into the genome of the HSPC forming a transcriptional unit that can express a receptor conferring a selective proliferation advantage on the genetically modified HSPC relative to the HSPC before modification.
A method of treating a subject, comprising
- (a) administering an immunotherapeutic agent specifically binding to c-Kit to deplete endogenous HSPCs expressing c-Kit;
- and
- (b) administering replacement HSPCs genetically modified to express a receptor conferring a selective proliferation advantage on the genetically modified HSPCs relative to endogenous HSPCs and thereby resist depletion by the immunotherapeutic agent specifically binding to c-Kit, wherein the replacement HSPCs at least partially replace the endogenous HSPCs.
Dependencies are inferred from the claim text.