Rescue of severely affected Dystrophin/Utrophin deficient mice through scAAV-U7snRNA mediated exon skipping: "In this study, we report for the first time the efficiency of the AAV-mediated exon-skipping approach in the utrophin/dystrophin double-knockout mouse (dKO) which is a very severe and progressive mouse model of DMD. Following a single intravenous injection of scAAV9-U7ex23 in dKO mice, near-normal levels of dystrophin expression were restored in all muscles examined, including the heart."
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