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Title:

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.

Document type:
Article; Early Access; Journal Article
Author(s):
Moretti, A; Fonteyne, L; Giesert, F; Hoppmann, P; Meier, A B; Bozoglu, T; Baehr, A; Schneider, C M; Sinnecker, D; Klett, K; Fröhlich, T; Rahman, F Abdel; Haufe, T; Sun, S; Jurisch, V; Kessler, B; Hinkel, R; Dirschinger, R; Martens, E; Jilek, C; Graf, A; Krebs, S; Santamaria, G; Kurome, M; Zakhartchenko, V; Campbell, B; Voelse, K; Wolf, A; Ziegler, T; Reichert, S; Lee, S; Flenkenthaler, F; Dorn, T; Jeremias, I; Blum, H; Dendorfer, A; Schnieke, A; Krause, S; Walter, M C; Klymiuk, N; Laugwitz, K L;...     »
Abstract:
Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene editing by sequence-specific nucleases offers new options for restoring the DMD reading frame, resulting in expression of a shortened but largely functional dystrophin protein. Here, we validated this approach in a pig model of DMD lacking exon 52 of DMD (DMDΔ52), as well as in a corresponding patient-derived induced pluripoten...     »
Journal title abbreviation:
Nat Med
Year:
2020
Journal volume:
26
Journal issue:
2
Pages contribution:
207-214
Fulltext / DOI:
doi:10.1038/s41591-019-0738-2
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/31988462
Print-ISSN:
1078-8956
TUM Institution:
Arbeitsgruppe Tissue Engineering und Regenerative Medizin; I. Medizinische Klinik und Poliklinik (Kardiologie)
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