Ontology highlight
ABSTRACT:
INSTRUMENT(S): Q Exactive
ORGANISM(S): Sus Scrofa Domesticus (domestic Pig)
TISSUE(S): Skeletal Muscle
DISEASE(S): Duchenne Muscular Dystrophy
SUBMITTER: Thomas Fröhlich
LAB HEAD: Thomas Fröhlich
PROVIDER: PXD014893 | Pride | 2020-02-05
REPOSITORIES: Pride
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OxidationMSites.txt | Txt | |||
allPeptides.txt | Txt | |||
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modificationSpecificPeptides.txt | Txt | |||
msScans.txt | Txt |
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Moretti A A Fonteyne L L Giesert F F Hoppmann P P Meier A B AB Bozoglu T T Baehr A A Schneider C M CM Sinnecker D D Klett K K Fröhlich T T Rahman F Abdel FA Haufe T T Sun S S Jurisch V V Kessler B B Hinkel R R Dirschinger R R Martens E E Jilek C C Graf A A Krebs S S Santamaria G G Kurome M M Zakhartchenko V V Campbell B B Voelse K K Wolf A A Ziegler T T Reichert S S Lee S S Flenkenthaler F F Dorn T T Jeremias I I Blum H H Dendorfer A A Schnieke A A Krause S S Walter M C MC Klymiuk N N Laugwitz K L KL Wolf E E Wurst W W Kupatt C C
Nature medicine 20200127 2
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 ...[more]