Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers

JCI insight - Tập 3 Số 9 - 2018
Eugene Wyatt1, Alexis R. Demonbreun1, Ellis Y. Kim2, Megan J. Puckelwartz1, Andy H. Vo3, Shoa L. Clarke1, Quan Q. Gao4, Mariz Vainzof5, R C M Pavanello5, Mayana Zatz5, Elizabeth M. McNally1
1Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago Illinois, USA
2Committee on Molecular Medicine and Molecular Pathogenesis and
3Committee on Developmental Biology and Regenerative Medicine, The University of Chicago, Chicago, Illinois, USA.
4Division of Hematology/Oncology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA
5Human Genome and stem-cell Center, Institute of Biosciences, University of São Paulo, São Paulo, Brazil

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