Niek P. van Til, Roya Sarwari, Trudi P. Visser, Julia Hauer, Chantal Lagresle‐Peyrou, Guus van der Velden, Vidyasagar Malshetty, Patricia Cortés, Arnaud Jollet, Olivier Danos, Barbara Cassani, Fang Zhang, Adrian J. Thrasher, Elena Fontana, Pietro Luigi Poliani, Marina Cavazzana, Monique M.A. Verstegen, Anna Villa, Gerard Wagemaker,
BackgroundRecombination-activating gene 1 (RAG1) deficiency results in severe combined immunodeficiency (SCID) caused by a complete lack of T and B lymphocytes. If untreated, patients succumb to recurrent infections.ObjectivesWe sought to develop lentiviral gene therapy for RAG1-induced SCID and to test its safety.MethodsConstructs containing the viral spleen-focus-forming virus (SF), ubiquitous promoters, or cell type-restricted promoters driving sequence-optimized RAG1 were compared for efficacy ...
Tópico(s): RNA Interference and Gene Delivery
2013 - Elsevier BV | Journal of Allergy and Clinical Immunology
Qiushi Liang, Merel Stok, Yvette van Helsdingen, Guus van der Velden, Ed Jacobs, Dirk J. Duncker, Arnold Reuser, Ans van der Ploeg, Arnold G. Vulto, Niek P. van Til, Gerard Wagemaker,
Pompe disease is a rare autosomal recessive metabolic disorder caused by defi ciency of lysosomal hydrolase acid α-glucosidase (GAA). GAA degrades glycogen to glucose, and defi ciency results in generalized tissue glycogen accumulation leading to cardiorespiratory failure in the early-onset patients within the fi rst year of life. Enzyme replacement therapy (ERT) by administration of recombinant acid α-glucosidase (alglucosidase alfa, Myozyme®) is currently the only effective treatment, requiring highdose ...
Tópico(s): Biochemical and Molecular Research
2015 - IOS Press | Journal of Neuromuscular Diseases
Ernest van Veen, Mathieu van der Jagt, Giuseppe Citerio, Nino Stocchetti, Diederik Gommers, Alex Burdorf, David Menon, Andrew I.R. Maas, Erwin J. O. Kompanje, Hester F. Lingsma, Cecilia Åkerlund, Krisztina Amrein, Nada Anđelić, Lasse Andreassen, Audny Anke, Anna Degli Antoni, Gérard Audibert, Philippe Azouvi, Maria Luisa Azzolini, Ronald Bartels, Pál Barzó, Romuald Beauvais, Ronny Beer, Bo‐Michael Bellander, Antonio Belli, Habib Benali, Maurizio Berardino, Luigi Beretta, Morten Blaabjerg, Peter Bragge, Alexandra Bražinová, Vibeke Brinck, Joanne Brooker, Camilla Brorsson, András Büki, Monika Bullinger, Manuel Cabeleira, Alessio Caccioppola, Emiliana Calappi, Maria Rosa Calvi, Peter Cameron, Guillermo Carbayo Lozano, Marco Carbonara, Simona Cavallo, Giorgio Chevallard, Arturo Chieregato, Giuseppe Citerio, Hans Clusmann, Mark Coburn, Jonathan Coles, D. James Cooper, Marta Correia, Amra Čović, Nicola Curry, Endre Czeiter, Marek Czosnyka, Claire Dahyot Fizelier, Paul Dark, Helen Dawes, Véronique De Keyser, Vincent Degos, Françesco Della Corte, Hugo den Boogert, Bart Depreitere, Đula Đilvesi, Abhishek Dixit, Emma Donoghue, Jens P. Dreier, Guy Loup Dulière, Ari Ercole, Patrick Esser, Erzsébet Ezer, Martin Fabricius, Valery L. Feigin, Kelly Foks, Shirin Frisvold, Alex Furmanov, Pablo Gagliardo, Damien Galanaud, Dashiell Gantner, Guoyi Gao, Pradeep George, Alexandre Ghuysen, Lelde Giga, Ben Glocker, Jagoš Golubović, Pedro A. Gómez, Johannes Gratz, Benjamin Gravesteijn, Francesca Grossi, Russell L. Gruen, Deepak Gupta, Juanita A. Haagsma, Iain Haitsma, Raimund Helbok, Eirik Helseth, Lindsay Horton, Jilske Huijben, Peter J. Hutchinson, Bram Jacobs, Stefan Jankowski, Mike Jarrett, Ji Y. Jiang, Faye Johnson, Kelly Jones, Mladen Karan, Angelos G. Kolias, Erwin J. O. Kompanje, Daniel Kondziella, Evgenios Kornaropoulos, Lars Owe Koskinen, Noémi Kovács, Ana Kowark, Alfonso Lagares, Linda Lanyon, Steven Laureys, Fiona Lecky, Didier Ledoux, Rolf Lefering, Valérie Legrand, Aurélie Lejeune, Leon Levi, Roger Lightfoot, Hester F. Lingsma, Andrew I.R. Maas, Ana M. Castaño‐León, Marc Maegele, Marek Majdán, Alex Manara, Geoffrey T. Manley, Costanza Martino, Hugues Maréchal, Julia Mattern, Catherine McMahon, Béla Melegh, David Menon, Tomas Menovsky, Ana Mikolić, Benoît Misset, Visakh Muraleedharan, Lynnette Murray, Ancuța Negru, David Nelson, Virginia Newcombe, Daan Nieboer, József Nyirádi, Otesile Olubukola, Matej Orešič, Fabrizio Ortolano, Aarno Palotie, Paul M. Parizel, Jean‐François Payen, Natascha Perera, Vincent Perlbarg, Paolo Persona, Wilco C. Peul, Anna Piippo-Karjalainen, Matti Pirinen, Dana Pisică, Horia Pleș, Suzanne Polinder, Iñigo Pomposo, Jussi P. Posti, Louis Puybasset, Andreea Rădoi, Arminas Ragauskas, Rahul Raj, Malinka Rambadagalla, Isabel Retel Helmrich, Jonathan R. Rhodes, Sylvia Richardson, Sophie Richter, Samuli Ripatti, Saulius Ročka, Cecilie Røe, Olav Røise, Jonathan Rosand, Jeffrey V. Rosenfeld, Christina Rosenlund, Guy Rosenthal, Rolf Rossaint, Sandra Rossi, Daniel Rueckert, Martin Rusňák, Juan Sahuquillo, Oliver Sakowitz, Renán Sánchez-Porras, János Sándor, Nadine Schäfer, Silke Schmidt, Herbert Schöechl, Guus Schoonman, Rico Frederik Schou, Elisabeth Schwendenwein, Charlie Sewalt, Toril Skandsen, Peter Smielewski, Abayomi Sorinola, Emmanuel A. Stamatakis, Simon Stanworth, Robert D. Stevens, William Stewart, Ewout W. Steyerberg, Nino Stocchetti, Nina Sundström, Riikka Takala, Viktória Tamás, Tomas Tamošuitis, Mark Steven Taylo, Braden Te Ao, Olli Tenovuo, Alice Theadom, Matt Thomas, Dick Tibboel, Marjolein Timmers, Christos Tolias, Tony Trapani, Cristina Maria Tudora, Andreas Unterberg, Peter Vajkoczy, Shirley Vallance, Egils Valeinis, Zoltán Vámos, Mathieu van der Jagt, Gregory Van der Steen, Joukje van der Naalt, Jeroen T.J.M. van Dijck, Thomas A. van Essen, Wim Van Hecke, Caroline van Heugten, Dominique Van Praag, Ernest van Veen, Thijs Vande Vyvere, Roel P. J. van Wijk, Alessia Vargiolu, Emmanuel Vega, Kimberley Velt, Jan Verheyden, Paul Vespa, Anne Vik, Rimantas Vilcinis, Victor Volovici, Nicole von Steinbüchel, Daphne Voormolen, Petar Vuleković, Kevin Wang, Eveline Wiegers, Guy Williams, Lindsay Wilson, Stefan Winzeck, Stefan Wolf, Zhihui Yang, Peter Ylén, Alexander Younsi, Frederick A. Zeiler, Veronika Zelinkova, Agate Ziverte, Tommaso Zoerle,
... group had a lower maximum therapy intensity level (TIL) score than patients in the later WLSM group ( ...
Tópico(s): Traumatic Brain Injury and Neurovascular Disturbances
2021 - Springer Science+Business Media | Intensive Care Medicine
A-L. Niemeijer, Egbert F. Smit, Idris Bahce, O. S. Hoekstra, M. Huisman, Guus AMS van Dongen, Albert D. Windhorst, N. Harry Hendrikse, Alex J. Poot, Daniëlle J. Vugts, David Leung, Wendy Hayes, R. Adam Smith, L. Wilson, Erik Thunnissen, J. De Langen,
... IHC (p = 0.03). For tumors with high TIL/stromal PD-1 expression, mean 89Zr-nivo SUVpeak ...
Tópico(s): Colorectal Cancer Treatments and Studies
2017 - Elsevier BV | Annals of Oncology
Shubhayu Bhattacharyay, Florian D. van Leeuwen, Erta Beqiri, Cecilia Åkerlund, Lindsay Wilson, Ewout W. Steyerberg, David Nelson, Andrew I.R. Maas, David Menon, Ari Ercole, Cecilia Åkerlund, Krisztina Amrein, Nada Anđelić, Lasse Andreassen, Audny Anke, Anna Degli Antoni, Gérard Audibert, Philippe Azouvi, Maria Luisa Azzolini, Ronald Bartels, Pál Barzó, Romuald Beauvais, Ronny Beer, Bo-Michael Bellander, Antonio Belli, Habib Benali, Maurizio Berardino, Luigi Beretta, Morten Blaabjerg, Peter Bragge, Alexandra Bražinová, Vibeke Brinck, Joanne Brooker, Camilla Brorsson, Andras Buki, Monika Bullinger, Manuel Cabeleira, Alessio Caccioppola, Emiliana Calappi, Maria Rosa Calvi, Peter Cameron, Guillermo Carbayo Lozano, Marco Carbonara, Simona Cavallo, Giorgio Chevallard, Arturo Chieregato, Giuseppe Citerio, Hans Clusmann, Mark Coburn, Jonathan Coles, D. James Cooper, Marta Correia, Amra Čović, Nicola Curry, Endre Czeiter, Marek Czosnyka, Claire Dahyot‐Fizelier, Paul Dark, Helen Dawes, Véronique De Keyser, Vincent Degos, Françesco Della Corte, Hugo den Boogert, Bart Depreitere, Đula Đilvesi, Abhishek Dixit, Emma Donoghue, Jens P. Dreier, Guy-Loup Dulière, Ari Ercole, Patrick Esser, Erzsébet Ezer, Martin Fabricius, Valery L. Feigin, Kelly Foks, Shirin Frisvold, Alex Furmanov, Pablo Gagliardo, Damien Galanaud, Dashiell Gantner, Guoyi Gao, Pradeep George, Alexandre Ghuysen, Lelde Giga, Ben Glocker, Jagoš Golubović, Pedro A. Gómez, Johannes Gratz, Benjamin Gravesteijn, Francesca Grossi, Russell L. Gruen, Deepak Gupta, Juanita A. Haagsma, Iain Haitsma, Raimund Helbok, Eirik Helseth, Lindsay Horton, Jilske Huijben, Peter J. Hutchinson, Bram Jacobs, Stefan Jankowski, Mike Jarrett, Jiyao Jiang, Faye Johnson, Kelly Jones, Mladen Karan, Angelos G. Kolias, Erwin J. O. Kompanje, Daniel Kondziella, Evgenios Kornaropoulos, Lars‐Owe Koskinen, Noémi Kovács, Ana Kowark, Alfonso Lagares, Linda Lanyon, Steven Laureys, Fiona Lecky, Didier Ledoux, Rolf Lefering, Valérie Legrand, Aurélie Lejeune, Leon Levi, Roger Lightfoot, Hester F. Lingsma, Andrew I.R. Maas, Ana M. Castaño‐León, Marc Maegele, Marek Majdán, Alex Manara, Geoffrey T. Manley, Costanza Martino, Hugues Maréchal, Julia Mattern, Catherine McMahon, Béla Melegh, David K. Menon, Tomas Menovsky, Ana Mikolić, Benoît Misset, Visakh Muraleedharan, Lynnette Murray, Ancuța Negru, David Nelson, Virginia Newcombe, Daan Nieboer, József Nyirádi, Otesile Olubukola, Matej Orešič, Fabrizio Ortolano, Aarno Palotie, Paul M. Parizel, Jean‐François Payen, Natascha Perera, Vincent Perlbarg, Paolo Persona, Wilco C. Peul, Anna Piippo-Karjalainen, Matti Pirinen, Dana Pisică, Horia Pleș, Suzanne Polinder, Iñigo Pomposo, Jussi P. Posti, Louis Puybasset, Andreea Rădoi, Arminas Ragauskas, Rahul Raj, Malinka Rambadagalla, Isabel Retel Helmrich, Jonathan R. Rhodes, Sylvia Richardson, Sophie Richter, Samuli Ripatti, Saulius Ročka, Cecilie Røe, Olav Røise, Jonathan Rosand, Jeffrey V. Rosenfeld, Christina Rosenlund, Guy Rosenthal, Rolf Rossaint, Sandra Rossi, Daniel Rueckert, Martin Rusňák, Juan Sahuquillo, Oliver Sakowitz, Renán Sánchez-Porras, János Sándor, Nadine Schäfer, Silke Schmidt, Herbert Schöechl, Guus Schoonman, Rico Frederik Schou, Elisabeth Schwendenwein, Charlie Sewalt, Ranjit D. Singh, Toril Skandsen, Peter Smielewski, Abayomi Sorinola, Emmanuel A. Stamatakis, Simon Stanworth, Robert D. Stevens, William Stewart, Ewout W. Steyerberg, Nino Stocchetti, Nina Sundström, Riikka Takala, Viktória Tamás, Tomas Tamošuitis, Mark Taylor, Aurore Thibaut, Braden Te Ao, Olli Tenovuo, Alice Theadom, Matt Thomas, Dick Tibboel, Marjolein Timmers, Christos Tolias, Tony Trapani, Cristina Maria Tudora, Andreas Unterberg, Peter Vajkoczy, Shirley Vallance, Egils Valeinis, Zoltán Vámos, Mathieu van der Jagt, Gregory Van der Steen, Joukje van der Naalt, Jeroen T.J.M. van Dijck, Inge A. M. van Erp, Thomas A. van Essen, Wim Van Hecke, Caroline van Heugten, Ernest van Veen, Thijs Vande Vyvere, Roel P. J. van Wijk, Alessia Vargiolu, Emmanuel Vega, Kimberley Velt, Jan Verheyden, Paul Vespa, Anne Vik, Rimantas Vilcinis, Victor Volovici, Nicole von Steinbüchel, Daphne Voormolen, Petar Vuleković, Kevin Wang, Daniel Whitehouse, Eveline Wiegers, Guy Williams, Stefan Winzeck, Stefan Wolf, Zhihui Yang, Peter Ylén, Alexander Younsi, Frederick A. Zeiler, Veronika Zelinkova, Agate Ziverte, Tommaso Zoerle,
... following day’s five-category therapy intensity level (TIL (Basic) ) score. With 20 repeats of fivefold cross- ... contributions towards predictions of next-day changes in TIL (Basic) . Based on Somers’ D xy , the full ... the ordinal variation in next-day changes in TIL (Basic) on day one and up to 51% (95% CI 45–56%) thereafter, when changes in TIL (Basic) became less frequent. Up to 81% (95% ... 79 [95% CI 0.76–0.82]) in TIL (Basic) after day two, we found specific predictor ... ordinal variation in day-to-day changes in TIL (Basic) after day two remained unexplained, underscoring the ...
Tópico(s): Trauma and Emergency Care Studies
2025 - Nature Portfolio | Scientific Reports
Anna-Larissa N. Niemeijer, Egbert F. Smit, Guus A.M.S. van Dongen, A. D. Windhorst, M. Huisman, N. Harry Hendrikse, Idris Bahce, D. Lueng, R. Smith, Wendy Hayes, Lori W. Wilson, Samuel J. Bonacorsi, Deirdre Donnelly, Patrice J. Morin, Alex J. Poot, Daniëlle J. Vugts, Erik Thunnissen, J. De Langen,
... IHC (p=0.03). For tumors with high TIL/ stromal PD-1 expression, mean 89Zr-nivo SUVpeak ...
Tópico(s): Medical Imaging Techniques and Applications
2017 - Elsevier BV | Journal of Thoracic Oncology