Universal donor cells
AssigneeCrispr Therapeutics AG
InventorsAlireza Rezania, Rebeca Ramos-Zayas
Life of this patent
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Abstract
Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.
Claims20
A method of generating a genetically modified stem cell, comprising
- (a) introducing an insertion of a polynucleotide encoding programmed death-ligand 1 (PD-L1) into the genome of a stem cell;
- (b) introducing an insertion of a polynucleotide encoding mesencephalic astrocyte derived neurotrophic factor (MANF) into the genome of the stem cell;
- and
- (c) introducing an insertion of a polynucleotide that encodes at least one safety switch into the genome of the cell,
- thereby generating a genetically modified stem cell expressing PD-L1 and MANF at an increased level relative to an unmodified stem cell.
Dependencies are inferred from the claim text.