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US 12,251,403 B2Granted
Genetically engineered hematopoietic stem cells and uses thereof
AssigneeVor Biopharma Inc.
InventorsJoseph Bolen, Aleksandar Filip Radovic-Moreno, John Lydeard
Life of this patent
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Abstract
Genetically engineered hematopoietic cells such as hematopoietic stem cells having one or more genetically edited genes of lineage-specific cell-surface proteins and therapeutic uses thereof, either alone or in combination with immune therapy that targets the lineage-specific cell-surface proteins.
Claims18
A genetically engineered human hematopoietic stem cell (HSC), or
- a descendant thereof, comprising a modified gene encoding a CD33 antigen and a modified gene encoding a CD123 antigen, wherein the modified gene encoding the CD33 antigen comprises a modified exon 2 or a modified exon 3 and wherein the modified gene encoding the CD123 antigen comprises a modified exon 5 or a modified exon 6;
- and reduced or eliminated expression of the CD33 antigen and the CD123 antigen relative to a wild-type human HSC.
Dependencies are inferred from the claim text.