Methods and compositions for treatment of a genetic condition
AssigneeSangamo Therapeutics, Inc.
InventorsGregory J. Cost, Dmitry Guschin, Philip D. Gregory, Michael C. Holmes, Jeffrey C. Miller, David Paschon, Edward J. Rebar, Andreas Reik, Fyodor Urnov, Lei Zhang
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Abstract
Methods and compositions for a genetic disease are provided.
Claims22
An isolated genetically modified mammalian cell comprising an insertion and/or deletion within an endogenous BCL11A gene made by a nuclease comprising a DNA-binding domain that binds to a target site as shown in any of SEQ ID NOs:56, 63, 66, 71, 160, 170, 179, 183, 189, 193, 197, 200, 203, 207, 211 and 213, such that the BCL11A gene is inactivated and expression of at least one globin gene in the cell is altered.
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Dependencies are inferred from the claim text.