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Three general strategies have been used to deliver ZFN in animal models. Upper, gene-modified offspring can be generated by directly injecting ZFN encoded DNA or mRNA into the embryos followed by implantation in foster mothers. Middle, human T cells or stem cells modified by ZFN ex vivo can then be transplanted to an animal model. Lower, ZFN can be delivered to the target cells to correct mutant genes by using adeno-associated virus (AAV) as a vector.
