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US 10,117,899 B2Granted
Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
AssigneesSangamo Therapeutics, Inc., Ospedale San Raffaele S.R.L., Fondazione Telethon
InventorsPietro Genovese, Philip D. Gregory, Michael C. Holmes, Angelo Leone Lombardo, Luigi Naldini
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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.
Claims11
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.
2 more elements
Dependencies are inferred from the claim text.