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Nuclease-mediated regulation of gene expression

  • US 10,619,154 B2
  • Filed: 03/21/2018
  • Issued: 04/14/2020
  • Est. Priority Date: 06/18/2015
  • Status: Active Grant
First Claim
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1. A method of altering globin gene expression in a cell, the method comprising:

  • introducing, into the cell, one or more polynucleotides encoding a zinc finger nuclease (ZFN) or a TAL-effector domain nuclease (TALEN) that binds to a target site in a BCL11A gene as shown in any of SEQ ID Nos;

    58-61, 65, 74 or 77, under conditions such that the nuclease cleaves and genetically modifies the BCL11A gene and expression of the globin gene is altered.

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