Litcius/Paper detail

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

Alessandra Moretti, Lina Marie Fonteyne, Florian Giesert, Petra Hoppmann, Anna B. Meier, Tarik Bozoglu, Andrea Baehr, Christine M. Schneider, Daniel Sinnecker, Katharina Klett, Thomas Fröhlich, Farah Abdel Rahman, Tobias Haufe, Shicheng Sun, Victoria Jurisch, Barbara Keßler, Rabea Hinkel, Ralf J. Dirschinger, Eimo Martens, Clemens Jilek, Alexander Graf, Stefan Krebs, Gianluca Santamaria, Mayuko Kurome, Valeri Zakhartchenko, Birgit Campbell, K. Voelse, Anja Wolf, Tilman Ziegler, Stefan Reichert, S. Lee, Florian Flenkenthaler, Tatjana Dorn, Irmela Jeremias, Helmut Blum, Andreas Dendorfer, Angelika Schnieke, Sabine Krause, Maggie C. Walter, Nikolai Klymiuk, Karl‐Ludwig Laugwitz, Eckhard Wolf, Wolfgang Wurst, Christian Kupatt

2020Nature Medicine236 citationsDOIOpen Access PDF

Topics & Concepts

Duchenne muscular dystrophyDystrophinInduced pluripotent stem cellBiologySkeletal muscleExon skippingMuscular dystrophyExonSomatic cellMyocyteCell biologyGeneticsGeneEmbryonic stem cellAlternative splicingAnatomyCRISPR and Genetic EngineeringVirus-based gene therapy researchMuscle Physiology and Disorders