Patent ID: 11911415
Assignee: EDITAS MEDICINE, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C  G | IPC A  C  G

Claim 0:
1. An in vitro or ex vivo method of reducing the cell surface expression of a protein encoded by a first allele of an endogenous human leukocyte antigen (HLA) gene in a blood cell, the method comprising:
contacting the blood cell with a first allele-specific gRNA molecule and a Cas9 molecule,
wherein the blood cell is isolated from a first subject having a first haplotype at the endogenous HLA gene,
wherein the first allele-specific gRNA molecule comprises a targeting domain which is complementary to a target domain in the HLA gene,
wherein the allele-specific gRNA molecule and the Cas9 molecule associate with and mediate a knock-out or knock-down of expression of the first allele of the endogenous HLA gene, and
wherein the first allele-specific gRNA molecule and the Cas9 molecule associate with a single allele of the HLA gene,
thereby reducing the cell surface expression of the protein encoded by the first allele of the endogenous HLA gene.