Benutzer: Gast  Login
Titel:

Pig models for translational Duchenne muscular dystrophy research.

Dokumenttyp:
Journal Article; Review
Autor(en):
Stirm, Michael; Klymiuk, Nikolai; Nagashima, Hiroshi; Kupatt, Christian; Wolf, Eckhard
Abstract:
Duchenne muscular dystrophy (DMD) is caused by mutations in the X-linked DMD gene, resulting in the absence of dystrophin, progressive muscle degeneration, and heart failure. Genetically tailored pig models resembling human DMD mutations recapitulate the biochemical, clinical, and pathological hallmarks of DMD with an accelerated disease progression compared to human patients. DMD pigs have been used to evaluate therapeutic concepts such as gene editing to reframe a disrupted DMD reading frame o...     »
Zeitschriftentitel:
Trends Mol Med
Jahr:
2024
Band / Volume:
30
Heft / Issue:
10
Seitenangaben Beitrag:
950-964
Volltext / DOI:
doi:10.1016/j.molmed.2024.04.013
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/38749865
Print-ISSN:
1471-4914
TUM Einrichtung:
Klinik und Poliklinik für Innere Medizin I, Kardiologie (Prof. Laugwitz); Professur für Kardiovaskuläre Translation in Großtiermodellen (Prof. Klymiuk)
 BibTeX