Restoring the regenerative balance in neuromuscular disorders: satellite cell activation as therapeutic target in Pompe disease

por Schaaf, Gerben J., Canibano-Fraile, Rodrigo, van Gestel, Tom J. M., van der Ploeg, Ans T., Pijnappel, W. W. M. Pim
Publicado no Ann Transl Med (2019)

Satellite cells maintain regenerative capacity but fail to repair disease-associated muscle damage in mice with Pompe disease

por Schaaf, Gerben J., van Gestel, Tom J. M., in ‘t Groen, Stijn L. M., de Jong, Bart, Boomaars, Björn, Tarallo, Antonietta, Cardone, Monica, Parenti, Giancarlo, van der Ploeg, Ans T., Pijnappel, W. W. M. Pim
Publicado no Acta Neuropathol Commun (2018)

GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells

por van der Wal, Erik, Bergsma, Atze J., van Gestel, Tom J.M., in ‘t Groen, Stijn L.M., Zaehres, Holm, Araúzo-Bravo, Marcos J., Schöler, Hans R., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Publicado no Mol Ther Nucleic Acids (2017)

Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies

por van der Wal, Erik, Herrero-Hernandez, Pablo, Wan, Raymond, Broeders, Mike, in 't Groen, Stijn L.M., van Gestel, Tom J.M., van IJcken, Wilfred F.J., Cheung, Tom H., van der Ploeg, Ans T., Schaaf, Gerben J., Pijnappel, W.W.M. Pim
Publicado no Stem Cell Reports (2018)