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US 2023/0227782 A1Published application
Hematopoietic stem and progenitor cells derived from hemogenic endothelial cells by episomal plasmid gene transfer
AssigneeChildren's Medical Center Corporation
InventorGeorge Q. DALEY
Life of this patent
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Abstract
Embodiments herein relate to in vitro production methods of hematopoietic stem cell (HSC) and hematopoietic stem and progenitor cell (HSPC) that have long-term multilineage hematopoiesis potentials upon in vivo engraftment. The HSC and HSPCs are derived from pluripotent stem cells-derived hemogenic endothelia cells (HE) by non-integrative episomal vectors-based gene transfer.
Claims16
A method of cellular replacement therapy in a subject in need thereof, the method comprising administering an engineered hetnatopoietic stem cells (HSCs) or hematopoietic stem and progenitor cells (HSPCs),
- wherein the engineered HSCs or HSPCs are produced by a method comprising in vitro transfecting hemogenic endothelia cells (HE) with an exogenous gene coding copy of at least one of the following transcription factors ERG, HOXA9, HOXA5, LCOR and RUNX1 comprised in a non-integrative vector,
- wherein the transcription factors are expressed in the transfeeted cells to produce a population of multilineage HSC and HSPCs that engrafts in recipient host after implantation.
Dependencies are inferred from the claim text.