Zobrazeno 1 - 10
of 16
pro vyhledávání: '"Sander J. van Deventer"'
Autor:
Josse A. Depla, Marina Sogorb-Gonzalez, Lance A. Mulder, Vivi M. Heine, Pavlina Konstantinova, Sander J. van Deventer, Katja C. Wolthers, Dasja Pajkrt, Adithya Sridhar, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 167-175 (2020)
The development of gene therapies for central nervous system disorders is challenging because it is difficult to translate preclinical data from current in vitro and in vivo models to the clinic. Therefore, we developed induced pluripotent stem cell
Externí odkaz:
https://doaj.org/article/6801bff942ae41f78c9545db8a90bbd9
Autor:
Sonay Keskin, Cynthia C. Brouwers, Marina Sogorb-Gonzalez, Raygene Martier, Josse A. Depla, Astrid Vallès, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 275-284 (2019)
Huntington disease (HD) is a fatal neurodegenerative genetic disorder, thought to reflect a toxic gain of function in huntingtin (Htt) protein. Adeno-associated viral vector serotype 5 (AAV5)- microRNA targeting huntingtin (miHTT) is a HD gene-therap
Externí odkaz:
https://doaj.org/article/ea22fa55f85944d198f8e622dca8256f
Autor:
Elisabeth A. Spronck, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula Montenegro-Miranda, Martin de Haan, Bart Nijmeijer, Valerie Ferreira, Harald Petry, Sander J. van Deventer
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 221-231 (2019)
Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5
Externí odkaz:
https://doaj.org/article/f68597eceef64b579989937b741d7460
Autor:
Kimberly L. Pietersz, Francois Du Plessis, Stephan M. Pouw, Jolanda M. Liefhebber, Sander J. van Deventer, Gerard J. M. Martens, Pavlina S. Konstantinova, Bas Blits
Publikováno v:
Frontiers in Bioengineering and Biotechnology, Vol 9 (2021)
Of the adeno-associated viruses (AAVs), AAV9 is known for its capability to cross the blood–brain barrier (BBB) and can, therefore, be used as a noninvasive method to target the central nervous system. Furthermore, the addition of the peptide PhP.B
Externí odkaz:
https://doaj.org/article/d366bbde948345e6abb55fdd9cea526b
Autor:
David Salas, Karin L. Kwikkers, Nerea Zabaleta, Andrea Bazo, Harald Petry, Sander J. van Deventer, Gloria Gonzalez Aseguinolaza, Valerie Ferreira
Publikováno v:
Blood Advances, Vol 3, Iss 17, Pp 2632-2641 (2019)
Abstract: Adeno-associated virus (AAV)–based liver gene therapy has been shown to be clinically successful. However, the presence of circulating neutralizing antibodies (NABs) against AAV vector capsids remains a major challenge as it may prevent s
Externí odkaz:
https://doaj.org/article/2914321f131b4a62a615768025d89710
Autor:
Elisabeth A. Spronck, Cynthia C. Brouwers, Astrid Vallès, Martin de Haan, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 334-343 (2019)
Huntington disease (HD) is a fatal neurodegenerative disorder caused by an autosomal dominant CAG repeat expansion in the huntingtin (HTT) gene. The translated expanded polyglutamine repeat in the HTT protein is known to cause toxic gain of function.
Externí odkaz:
https://doaj.org/article/80df46c476b84f378c89927542f2b137
Autor:
Raygene Martier, Jolanda M. Liefhebber, Ana García-Osta, Jana Miniarikova, Mar Cuadrado-Tejedor, Maria Espelosin, Susana Ursua, Harald Petry, Sander J. van Deventer, Melvin M. Evers, Pavlina Konstantinova
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 16, Iss , Pp 26-37 (2019)
A hexanucleotide GGGGCC expansion in intron 1 of chromosome 9 open reading frame 72 (C9orf72) gene is the most frequent cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The corresponding repeat-containing sense and anti
Externí odkaz:
https://doaj.org/article/7205c963663e4645b6377c073cfd2e0c
Autor:
Raygene Martier, Jolanda M. Liefhebber, Jana Miniarikova, Tom van der Zon, Jolanda Snapper, Iris Kolder, Harald Petry, Sander J. van Deventer, Melvin M. Evers, Pavlina Konstantinova
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 14, Iss , Pp 593-608 (2019)
The most common pathogenic mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is an intronic GGGGCC (G4C2) repeat in the chromosome 9 open reading frame 72 (C9orf72) gene. Cellular toxicity due to RNA foci and dipeptide
Externí odkaz:
https://doaj.org/article/84305642532d46c7a5bc8e546a17f209
Autor:
Elisabeth A. Spronck, Astrid Vallès, Margit H. Lampen, Paula S. Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M. Evers, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Martin de Haan
Publikováno v:
Brain Sciences, Vol 11, Iss 2, p 129 (2021)
Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism. There are currently no disease modifying therapies for HD; however, HTT-loweri
Externí odkaz:
https://doaj.org/article/003bb3f8cb854143be21f0cb86c10923
Autor:
Wouter J. de Jonge, Frans O. The, Dennis van der Coelen, Roelof J. Bennink, Pieter H. Reitsma, Sander J. van Deventer, René M. Van den Wijngaard, Guy E. Boeckxstaens
Publikováno v:
Gastroenterology, 127(2), 535-545. W.B. Saunders Ltd
Background & Aims : Inflammation of the intestinal muscularis following manipulation during surgery plays a crucial role in the pathogenesis of postoperative ileus. Here, we evaluate the role of mast cell activation in the recruitment of infiltrates
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5f57e3f30777dce565c7142c686e6558
https://doi.org/10.1053/j.gastro.2004.04.017
https://doi.org/10.1053/j.gastro.2004.04.017