×

Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells

  • US 10,117,899 B2
  • Filed: 10/16/2014
  • Issued: 11/06/2018
  • Est. Priority Date: 10/17/2013
  • Status: Active Grant
First Claim
Patent Images

1. A method for targeted integration into purified hematopoietic stem cells (HSC) and/or progenitor cells (PC), the method comprising the following steps:

  • (a) stimulating the cells;

    (b) delivering a non-integrating lentiviral (IDLV) donor nucleic acid to the cells one day after stimulation;

    (c) culturing the cells obtained by step (b) for one day; and

    (d) delivering at least one zinc finger nuclease to the cells obtained by step (c) such that the donor nucleic acid is integrated into the genome, wherein the at least one zinc finger nuclease is delivered in mRNA form.

View all claims
  • 3 Assignments
Timeline View
Assignment View
    ×
    ×