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Genetically modified human cell with a corrected mutant sickle cell mutation

  • US 10,435,677 B2
  • Filed: 09/16/2015
  • Issued: 10/08/2019
  • Est. Priority Date: 09/16/2014
  • Status: Active Grant
First Claim
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1. An isolated genetically modified human cell comprising a pair of zinc finger nucleases (ZFNs), the pair comprising the zinc finger proteins (ZFPs) designated 47773 and 47817, wherein 47773 binds to a target site within SEQ ID NO:

  • 23 and 47817 binds to a SEQ ID NO;

    24 of an endogenous human beta-hemoglobin (Hbb) gene comprising a sickle cell mutation, wherein the pair of ZFNs makes a genomic modification in the mutant endogenous Hbb gene and the genomic modification comprises insertion of a donor sequence such that the sickle cell mutation is corrected to wild-type Hbb, and the cell expresses wild-type Hbb.

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