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Department of Medical Genetics
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LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts.
Scientific reports Jun, 2017 | Pubmed ID: 28623256
University of Alberta Faculty of Medicine and Dentistry
Rika Maruyama1,
Aleksander Touznik1,
Toshifumi Yokota1,2
1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry,
2Muscular Dystrophy Canada Research Chair, Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry
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