Bioengineered constructs combined with exercise enhance stem cell-mediated treatment of volumetric muscle loss
Quarta, Marco, Cromie, Melinda, Chacon, Robert, Blonigan, Justin, Garcia, Victor, Akimenko, Igor, Hamer, Mark, Paine, Patrick, Stok, Merel, Shrager, Joseph B, Rando, Thomas A
Published in Nature communications (20.06.2017)
Published in Nature communications (20.06.2017)
Get full text
Journal Article
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
Visigalli, Ilaria, Delai, Stefania, Politi, Letterio S., Di Domenico, Carmela, Cerri, Federica, Mrak, Emanuela, D'Isa, Raffaele, Ungaro, Daniela, Stok, Merel, Sanvito, Francesca, Mariani, Elisabetta, Staszewsky, Lidia, Godi, Claudia, Russo, Ilaria, Cecere, Francesca, del Carro, Ubaldo, Rubinacci, Alessandro, Brambilla, Riccardo, Quattrini, Angelo, Di Natale, Paola, Ponder, Katherine, Naldini, Luigi, Biffi, Alessandra
Published in Blood (09.12.2010)
Published in Blood (09.12.2010)
Get full text
Journal Article
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy
Liang, Qiushi, Catalano, Fabio, Vlaar, Eva C., Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., van der Ploeg, Ans T., van Til, Niek P., Pijnappel, W.W.M. Pim
Published in Molecular therapy. Methods & clinical development (08.12.2022)
Published in Molecular therapy. Methods & clinical development (08.12.2022)
Get full text
Journal Article
Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease
Stok, Merel, de Boer, Helen, Huston, Marshall W., Jacobs, Edwin H., Roovers, Onno, Visser, Trudi P., Jahr, Holger, Duncker, Dirk J., van Deel, Elza D., Reuser, Arnold J.J., van Til, Niek P., Wagemaker, Gerard
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Get full text
Journal Article
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
Liang, Qiushi, Vlaar, Eva C., Catalano, Fabio, Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., Unger, Wendy W.J., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim, van Til, Niek P.
Published in Molecular therapy. Methods & clinical development (09.06.2022)
Published in Molecular therapy. Methods & clinical development (09.06.2022)
Get full text
Journal Article
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype
van Til, Niek P., Stok, Merel, Aerts Kaya, Fatima S.F., de Waard, Monique C., Farahbakhshian, Elnaz, Visser, Trudi P., Kroos, Marian A., Jacobs, Edwin H., Willart, Monique A., van der Wegen, Pascal, Scholte, Bob J., Lambrecht, Bart N., Duncker, Dirk J., van der Ploeg, Ans T., Reuser, Arnold J.J., Verstegen, Monique M., Wagemaker, Gerard
Published in Blood (01.07.2010)
Published in Blood (01.07.2010)
Get full text
Journal Article
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE
Yadak, Rana, Cabrera-Pérez, Raquel, Torres-Torronteras, Javier, Bugiani, Marianna, Haeck, Joost C, Huston, Marshall W, Bogaerts, Elly, Goffart, Steffi, Jacobs, Edwin H, Stok, Merel, Leonardelli, Lorena, Biasco, Luca, Verdijk, Robert M, Bernsen, Monique R, Ruijter, George, Martí, Ramon, Wagemaker, Gerard, van Til, Niek P, de Coo, Irenaeus F M
Published in Molecular therapy. Methods & clinical development (16.03.2018)
Published in Molecular therapy. Methods & clinical development (16.03.2018)
Get full text
Journal Article
Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy
de Vries, Juna M., Kuperus, Esther, Hoogeveen-Westerveld, Marianne, Kroos, Marian A., Wens, Stephan C.A., Stok, Merel, van der Beek, Nadine A.M.E., Kruijshaar, Michelle E., Rizopoulos, Dimitris, van Doorn, Pieter A., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in Genetics in medicine (01.01.2017)
Published in Genetics in medicine (01.01.2017)
Get full text
Journal Article
Response to Herbert et al
de Vries, Juna M, Kuperus, Esther, Hoogeveen-Westerveld, Marianne, Kroos, Marian A, Wens, Stephan C A, Stok, Merel, van der Beek, Nadine A M E, Kruijshaar, Michelle E, Rizopoulos, Dimitris, van Doorn, Pieter A, van der Ploeg, Ans T, Pijnappel, W W M Pim
Published in Genetics in medicine (01.11.2017)
Published in Genetics in medicine (01.11.2017)
Get full text
Journal Article
Lentiviral Stem Cell Gene Therapy for Pompe Disease
Liang, Qiushi, Stok, Merel, van Helsdingen, Yvette, van der Velden, Guus, Jacobs, Ed, Duncker, Dirk, Reuser, Arnold, van der Ploeg, Ans, Vulto, Arnold, van Til, Niek P, Wagemaker, Gerard
Published in Journal of neuromuscular diseases (2015)
Published in Journal of neuromuscular diseases (2015)
Get more information
Journal Article