DELIVERY METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED GENOME ENGINEERING IN HEMATOPOIETIC STEM CELLS
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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Abstract
The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.
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Citations
16 Claims
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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:
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(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. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16)
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Specification