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Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
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机译:大范围核酸酶试剂在治疗由移码/非正义突变引起的遗传疾病中的用途
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摘要
The present invention relates to a method to treat a genetic disease in an individual caused by at least one frame shift or at least one non sense mutation in the human dystrophin gene comprising at least the step of bringing into contact at least one meganuclease enzyme, which recognizes and cuts a target site in the human dystrophin gene, with the genome of said individual under conditions wherein said at least one meganuclease recognizes and cleaves its target site in the human dystrophin gene. Said method applies also to a set of meganuclease enzymes, which each recognizes and cuts a different target site. The present invention also relates to a kit comprising, at least one meganuclease enzyme as defined above and medicament comprising said meganuclease.
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