Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
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United States of America Patent
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Aug 12, 2014
Grant Date -
Nov 29, 2012
app pub date -
Sep 24, 2010
filing date -
Sep 24, 2009
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Abstract
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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