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Title:

A scalable, clinically severe pig model for Duchenne muscular dystrophy.

Document type:
Journal Article; Research Support, Non-U.S. Gov't
Author(s):
Stirm, Michael; Fonteyne, Lina Marie; Shashikadze, Bachuki; Lindner, Magdalena; Chirivi, Maila; Lange, Andreas; Kaufhold, Clara; Mayer, Christian; Medugorac, Ivica; Kessler, Barbara; Kurome, Mayuko; Zakhartchenko, Valeri; Hinrichs, Arne; Kemter, Elisabeth; Krause, Sabine; Wanke, Rüdiger; Arnold, Georg J; Wess, Gerhard; Nagashima, Hiroshi; Hrabĕ de Angelis, Martin; Flenkenthaler, Florian; Kobelke, Levin Arne; Bearzi, Claudia; Rizzi, Roberto; Bähr, Andrea; Reese, Sven; Matiasek, Kaspar; Walter, Ma...     »
Abstract:
Large-animal models for Duchenne muscular dystrophy (DMD) are crucial for the evaluation of diagnostic procedures and treatment strategies. Pigs cloned from male cells lacking DMD exon 52 (DMDΔ52) exhibit molecular, clinical and pathological hallmarks of DMD, but die before sexual maturity and cannot be propagated by breeding. Therefore, we generated female DMD+/- carriers. A single founder animal had 11 litters with 29 DMDY/-, 34 DMD+/- as well as 36 male and 29 female wild-type offspring. Bree...     »
Journal title abbreviation:
Dis Model Mech
Year:
2021
Journal volume:
14
Journal issue:
12
Fulltext / DOI:
doi:10.1242/dmm.049285
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/34796900
Print-ISSN:
1754-8403
TUM Institution:
1693; 617; Klinik und Poliklinik für Innere Medizin I, Kardiologie
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