Correction du gène de la dystrophine avec les nucléases à doigts de zinc
|Abstract:||Gene therapy without gene transfer using specific restriction endonucleases is a therapeutic approaches aimed at the development of a cure for Duchenne muscular dystrophy (DMD). To correct the dystrophin gene with zinc finger nucleases (ZFNs) targeting exon 50of DYS gene, we produced ZFNs proteins in bacteria and purified them. The results obtained after in vitro assays show that ZFNs produced specifically recognize a target sequence located in exon 50 of the gene DYS and can be generated in a precise manner the double strand breaks. They also show that ZFNs produced proteins can be transduced with or without agent transduction, in cultured myoblasts of patients’ Duchenne dystrophy.|
|Document Type:||Mémoire de maîtrise|
|Open Access Date:||19 April 2018|
|Collection:||Thèses et mémoires|
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