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Title:

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

Document type:
Journal Article; Review
Author(s):
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...     »
Journal title abbreviation:
Neuromuscul Disord
Year:
2022
Journal volume:
32
Journal issue:
7
Pages contribution:
543-556
Fulltext / DOI:
doi:10.1016/j.nmd.2022.04.005
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/35659494
Print-ISSN:
0960-8966
TUM Institution:
1693; Klinik und Poliklinik für Innere Medizin I, Kardiologie
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