Genetic modification of hepatocytes
AssigneeBeam Therapeutics Inc.
InventorsGiuseppe CIARAMELLA, Jason Michael GEHRKE, Ryan MURRAY
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Abstract
The present invention provides methods of producing genetically modified human hepatocytes suitable for hepatocyte transplantation comprising: disrupting one or more major histocompatibility complex (MHC) Class I or Class II genes in isolated human hepatocytes or in a hepatocyte progenitor cell by introducing a base editor and one or more gRNAs that hybridize with a target sequence in the one or more Class I or Class II genes, thereby producing genetically modified human hepatocytes.
Claims35
A method of producing genetically modified human hepatocytes suitable for hepatocyte transplantation comprising:
- disrupting one or more major histocompatibility complex (MHC) Class I or Class II genes in isolated human hepatocytes or in a hepatocyte progenitor cell by introducing a base editor and one or more gRNAs that hybridize with a target sequence in the one or more Class I or Class II genes, thereby producing genetically modified human hepatocytes.
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A eukaryotic cell comprising the base editor and one or more gRNAs comprising any one of the sequences listed in Tables 2A-6A or an RNA version of any one of the protospacer sequences listed in Table 2-6 that hybridize with a target sequence, wherein the cell is a human hepatocyte.
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A guide RNA comprising any one of the sequences listed in Tables 2A-6A or an RNA version of any one of the protospacer sequences listed in Table 2-6, wherein one, two, three, or more than three edits are made to the target gene.
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Dependencies are inferred from the claim text.