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Titel:

Pig models for Duchenne muscular dystrophy - from disease mechanisms to validation of new diagnostic and therapeutic concepts.

Dokumenttyp:
Journal Article; Review
Autor(en):
Stirm, Michael; Fonteyne, Lina Marie; Shashikadze, Bachuki; Stöckl, Jan B; Kurome, Mayuko; Keßler, Barbara; Zakhartchenko, Valeri; Kemter, Elisabeth; Blum, Helmut; Arnold, Georg J; Matiasek, Kaspar; Wanke, Rüdiger; Wurst, Wolfgang; Nagashima, Hiroshi; Knieling, Ferdinand; Walter, Maggie C; Kupatt, Christian; Fröhlich, Thomas; Klymiuk, Nikolai; Blutke, Andreas; Wolf, Eckhard
Abstract:
Duchenne muscular dystrophy (DMD) is a fatal X-linked disease caused by mutations in the DMD gene, leading to complete absence of dystrophin and progressive degeneration of skeletal muscles and heart. Animal models are essential for preclinical evaluation of novel diagnostic procedures and treatment strategies. Gene targeting/editing offers the possibility of developing tailored pig models for monogenic diseases. The first porcine DMD model was generated by deletion of DMD exon 52 (DMDΔ52) in cu...     »
Zeitschriftentitel:
Neuromuscul Disord
Jahr:
2022
Band / Volume:
32
Heft / Issue:
7
Seitenangaben Beitrag:
543-556
Volltext / DOI:
doi:10.1016/j.nmd.2022.04.005
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/35659494
Print-ISSN:
0960-8966
TUM Einrichtung:
1693; Klinik und Poliklinik für Innere Medizin I, Kardiologie
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