Cd38 as integration site for enhanced function of gene-modified immune cells
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsDean LEE, Meisam NAEIMI KARAROUDI
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Abstract
Disclosed herein is a genetically engineered cells expressing a chimeric antigen receptor and methods and uses thereof.
Claims28
An engineered cell comprising a nucleic acid sequence encoding a chimeric antigen receptor (CAR) comprising a single-chain variable fragment (scFV) that specifically binds to a target molecule, wherein the nucleic acid sequence encoding the CAR is integrated into a CD38 gene locus, and wherein the engineered cell has a decreased expression of CD38 as compared with a reference control.
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11-12. (canceled)
A pharmaceutical composition comprising an engineered cell and a pharmaceutically acceptable carrier, wherein the engineered cell comprises a nucleic acid sequence encoding a chimeric antigen receptor (CAR) comprising a single-chain variable fragment (scFV) that specifically binds to a target molecule, wherein the nucleic acid sequence encoding the CAR is integrated into a CD38 gene locus, and wherein the engineered cell has a decreased expression of CD38 as compared with a reference control.
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A method of treating a disease in a subject comprising administering to the subject a therapeutically effective amount of a engineered cell or a pharmaceutical composition comprising the engineered cell and a pharmaceutically acceptable carrier, wherein the engineered cell comprises a nucleic acid sequence encoding a chimeric antigen receptor (CAR) comprising a single-chain variable fragment (scFV) that specifically binds to a target molecule, wherein the nucleic acid sequence encoding the CAR is integrated into a CD38 gene locus, wherein the engineered cell has a decreased expression of CD38 as compared with a reference control.
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Dependencies are inferred from the claim text.