Zobrazeno 1 - 10
of 57
pro vyhledávání: '"Niek P, van Til"'
Autor:
Bonnie C. Plug, Ilma M. Revers, Marjolein Breur, Gema Muñoz González, Jaap A. Timmerman, Niels R.C. Meijns, Daniek Hamberg, Jikke Wagendorp, Erik Nutma, Nicole I. Wolf, Antonio Luchicchi, Huibert D. Mansvelder, Niek P. van Til, Marjo S. van der Knaap, Marianna Bugiani
Publikováno v:
Acta Neuropathologica Communications, Vol 12, Iss 1, Pp 1-17 (2024)
Abstract Human brain experimental models recapitulating age- and disease-related characteristics are lacking. There is urgent need for human-specific tools that model the complex molecular and cellular interplay between different cell types to assess
Externí odkaz:
https://doaj.org/article/95f2002b28a24aafa6da02213860ae53
Autor:
Zsolt Sebestyen, Jürgen Kuball, Maud Plantinga, Anja Krippner-Heidenreich, S Nierkens, Ester Dunnebach, Vania Lo Presti, Olaf T Heidenreich, Angelo Meringa, Alice van Velzen, Aida Valera Moreira, Ronald W Stam, Rishi S Kotecha, Annelisa Cornel, Niek P van Til
Publikováno v:
Journal for ImmunoTherapy of Cancer, Vol 12, Iss 4 (2024)
Background Hematopoietic cell transplantation (HCT) is an effective treatment for pediatric patients with high-risk, refractory, or relapsed acute myeloid leukemia (AML). However, a large proportion of transplanted patients eventually die due to rela
Externí odkaz:
https://doaj.org/article/201ace7f9fc54b5eb4254af5c2cc872c
Autor:
Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Tim Maiwald, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 464-487 (2022)
Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly i
Externí odkaz:
https://doaj.org/article/19aee40652b94df1a4979d0e41238f5c
Autor:
Qiushi Liang, Fabio Catalano, Eva C. Vlaar, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Ans T. van der Ploeg, Niek P. van Til, W.W.M. Pim Pijnappel
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 109-130 (2022)
Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen accumulation in various tissues, including cardiac and skeletal muscles and the central nervous system (CNS). Enzyme replacement therapy (ERT) improves cardiac,
Externí odkaz:
https://doaj.org/article/310576551caf4a2592416b696428e590
Autor:
Qiushi Liang, Eva C. Vlaar, Fabio Catalano, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Wendy W.J. Unger, Ans T. van der Ploeg, W.W.M. Pim Pijnappel, Niek P. van Til
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 520-532 (2022)
Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). ERT has shown to be lifesaving in patients with classic infa
Externí odkaz:
https://doaj.org/article/833c3541c3c74a63aa8c8bff6fa7155d
Autor:
Fatima Aerts-Kaya, Niek P. van Til
Publikováno v:
Pharmaceutics, Vol 15, Iss 11, p 2522 (2023)
Leukodystrophies are a heterogenous group of inherited, degenerative encephalopathies, that if left untreated, are often lethal at an early age. Although some of the leukodystrophies can be treated with allogeneic hematopoietic stem cell transplantat
Externí odkaz:
https://doaj.org/article/ca0f9b3814034bc8a610a1e73c752ec3
Autor:
Vania Lo Presti, Annelisa M. Cornel, Maud Plantinga, Ester Dünnebach, Jurgen Kuball, Jaap Jan Boelens, Stefan Nierkens, Niek P. van Til
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 357-368 (2021)
Adoptive T cell therapy utilizing tumor-specific autologous T cells has shown promising results for cancer treatment. However, the limited numbers of autologous tumor-associated antigen (TAA)-specific T cells and the functional aberrancies, due to di
Externí odkaz:
https://doaj.org/article/437a3fa6e3444d1fa4601d5b93d4d03b
Autor:
Merel Stok, Helen de Boer, Marshall W. Huston, Edwin H. Jacobs, Onno Roovers, Trudi P. Visser, Holger Jahr, Dirk J. Duncker, Elza D. van Deel, Arnold J.J. Reuser, Niek P. van Til, Gerard Wagemaker
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 1014-1025 (2020)
Pompe disease is an autosomal recessive lysosomal storage disorder characterized by progressive muscle weakness. The disease is caused by mutations in the acid α-glucosidase (GAA) gene. Despite the currently available enzyme replacement therapy (ERT
Externí odkaz:
https://doaj.org/article/81835f573420466a90338ee21c4f9707
Publikováno v:
Biomedicines, Vol 10, Iss 2, p 302 (2022)
Pompe disease is an inherited neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset Pompe disease, presenting shortly after birth with symptoms of cardiomyopathy, res
Externí odkaz:
https://doaj.org/article/c76acd8f3abf4980b14f9ce876629d2b
Autor:
Rana Yadak, Max V. Boot, Niek P. van Til, Dominique Cazals-Hatem, Armin Finkenstedt, Elly Bogaerts, Irenaeus F. de Coo, Marianna Bugiani
Publikováno v:
BMC Gastroenterology, Vol 18, Iss 1, Pp 1-6 (2018)
Abstract Background Gastrointestinal complications are the main cause of death in patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). Available treatments often restore biochemical homeostasis, but fail to cure gastrointestin
Externí odkaz:
https://doaj.org/article/d086ac5e51a34af49fc629842d942533