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DELIVERY METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED GENOME ENGINEERING IN HEMATOPOIETIC STEM CELLS

  • US 20150174169A1
  • Filed: 10/16/2014
  • Published: 06/25/2015
  • Est. Priority Date: 10/17/2013
  • Status: Active Grant
First Claim
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1. A method for targeted integration into purified hematopoietic stem cells (HSC) and/or progenitor cells (PC), said method comprising the following steps:

  • (a) delivering a non-integrating lentiviral (IDLV) donor nucleic acid to the HSC and/or PC cells;

    (b) culturing the cells obtained by step (a); and

    (c) delivering at least one zinc finger nuclease to the cells obtained by step (b) such that the donor nucleic acid is integrated into the genome.

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