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AssigneeCellectis
InventorsJacques Tremblay, Frederic Cedrone
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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.
A method of cleaving a human dystrophin gene comprising administering to an isolated cell comprising a human dystrophin gene at least one meganuclease polypeptide, which recognizes and cleaves a target site in the human dystrophin gene, wherein said meganuclease polypeptide comprises an amino acid sequence selected from the group consisting of SEQ ID NOs:
Dependencies are inferred from the claim text.