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Project context and summary :
Many neurodegenerative disorders are caused by the large expansion of a repeated sequence, called a "trinucleotide repeat". Our laboratory is using the CRISPR-Cas family of endonucleases in order to shorten the repeat tract below the length that is known to be pathological in humans. Some of the nucleases tested are very efficient at cutting the repeat tract, but in order to make this approach a viable gene therapy strategy, we must ensure that the nuclease is not inducing mutations in other parts of the genome (so-called "off-targets"). The present project aims at analyzing all possible off-targets of these nucleases on the different repeated sequences involved in neurodegenerative disorders, in order to validate (or invalidate) this approach.Related team publications :
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