Zobrazeno 1 - 10
of 57
pro vyhledávání: '"Niek P van Til"'
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:
Elnaz Farahbakhshian, Monique M Verstegen, Trudi P Visser, Sima Kheradmandkia, Dirk Geerts, Shazia Arshad, Noveen Riaz, Frank Grosveld, Niek P van Til, Jules P P Meijerink
Publikováno v:
PLoS ONE, Vol 9, Iss 8, p e105642 (2014)
Allogeneic hematopoietic stem cell (HSC) transplantations from umbilical cord blood or autologous HSCs for gene therapy purposes are hampered by limited number of stem cells. To test the ability to expand HSCs in vitro prior to transplantation, two g
Externí odkaz:
https://doaj.org/article/582a3ab15d1841a4aa688c9dadabc450
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:
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:
Robert N. Plasschaert, Mark P. DeAndrade, Fritz Hull, Quoc Nguyen, Tara Peterson, Aimin Yan, Mariana Loperfido, Cristina Baricordi, Luigi Barbarossa, John K. Yoon, Yildirim Dogan, Zeenath Unnisa, Jeffrey W. Schindler, Niek P. van Til, Luca Biasco, Chris Mason
Publikováno v:
Molecular Therapy. 30:3209-3225
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological benefit in rare diseases, which is achieved through the engraftment of genetically modified microglia-like cells (MLCs) in the brain. Still, the engraftment dynami
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ad5df9c34055a087315833104af0a261
https://doi.org/10.1016/j.ymthe.2022.05.022
https://doi.org/10.1016/j.ymthe.2022.05.022
Publikováno v:
Vaccines, Vol 8, Iss 4, p 733 (2020)
Recent developments in gene engineering technologies have drastically improved the therapeutic treatment options for cancer patients. The use of effective chimeric antigen receptor T (CAR-T) cells and recombinant T cell receptor engineered T (rTCR-T)
Externí odkaz:
https://doaj.org/article/7da909edf5be4d578f365f3e6f5cef2e