Murine CD4(+)CD25(-) cells activated in vitro with PMA/ionomycin and anti-CD3 acquire regulatory function and ameliorate experimental colitis in vivo

por Majowicz, Anna, van der Marel, Sander, te Velde, Anje A, Meijer, Sybren L, Petry, Harald, van Deventer, Sander J, Ferreira, Valerie
Publicado 2012

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

por Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

Genetically Modified Lactococcus lactis for Delivery of Human Interleukin-10 to Dendritic Cells

por Huibregtse, Inge L., Zaat, Sebatian A., Kapsenberg, Martien L., Sartori da Silva, Maria A., Peppelenbosch, Maikel P., van Deventer, Sander J. H., Braat, Henri
Publicado 2012

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

por Majowicz, Anna, Nijmeijer, Bart, Lampen, Margit H., Spronck, Lisa, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Meyer, Christian, Tangelder, Marco, Ferreira, Valerie
Publicado 2019

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

por Keskin, Sonay, Brouwers, Cynthia C., Sogorb-Gonzalez, Marina, Martier, Raygene, Depla, Josse A., Vallès, Astrid, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Publicado 2019

PhP.B Enhanced Adeno-Associated Virus Mediated-Expression Following Systemic Delivery or Direct Brain Administration

por Pietersz, Kimberly L., Plessis, Francois Du, Pouw, Stephan M., Liefhebber, Jolanda M., van Deventer, Sander J., Martens, Gerard J. M., Konstantinova, Pavlina S., Blits, Bas
Publicado 2021

Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

por Martier, Raygene, Liefhebber, Jolanda M., Miniarikova, Jana, van der Zon, Tom, Snapper, Jolanda, Kolder, Iris, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Publicado 2019

Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy

por Martier, Raygene, Liefhebber, Jolanda M., García-Osta, Ana, Miniarikova, Jana, Cuadrado-Tejedor, Mar, Espelosin, Maria, Ursua, Susana, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Publicado 2019

Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

por Spronck, Elisabeth A., Liu, Ying Poi, Lubelski, Jacek, Ehlert, Erich, Gielen, Sander, Montenegro-Miranda, Paula, de Haan, Martin, Nijmeijer, Bart, Ferreira, Valerie, Petry, Harald, van Deventer, Sander J.
Publicado 2019

Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain

por Depla, Josse A., Sogorb-Gonzalez, Marina, Mulder, Lance A., Heine, Vivi M., Konstantinova, Pavlina, van Deventer, Sander J., Wolthers, Katja C., Pajkrt, Dasja, Sridhar, Adithya, Evers, Melvin M.
Publicado 2020

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

por Spronck, Elisabeth A., Vallès, Astrid, Lampen, Margit H., Montenegro-Miranda, Paula S., Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M., Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, de Haan, Martin
Publicado 2021

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

por Miniarikova, Jana, Zanella, Ilaria, Huseinovic, Angelina, van der Zon, Tom, Hanemaaijer, Evelyn, Martier, Raygene, Koornneef, Annemart, Southwell, Amber L, Hayden, Michael R, van Deventer, Sander J, Petry, Harald, Konstantinova, Pavlina
Publicado 2016

Longitudinal Assessment of Lipoprotein(a) Levels in Perinatally HIV-Infected Children and Adolescents

por van Genderen, Jason G., Van den Hof, Malon, de Boer, Claudia G., Jansen, Hans P. G., van Deventer, Sander J. H., Tsimikas, Sotirios, Witztum, Joseph L., Kastelein, John J. P., Pajkrt, Dasja
Publicado 2021

Elevated Lipoprotein(a) in Perinatally HIV-Infected Children Compared With Healthy Ethnicity-Matched Controls

por Van den Hof, Malon, Klein Haneveld, Mirthe J, Blokhuis, Charlotte, Scherpbier, Henriette J, Jansen, Hans P G, Kootstra, Neeltje A, Dallinga-Thie, Geesje M, Van Deventer, Sander J H, Tsimikas, Sotirios, Pajkrt, Dasja
Publicado 2019

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

por Caron, Nicholas S, Southwell, Amber L, Brouwers, Cynthia C, Cengio, Louisa Dal, Xie, Yuanyun, Black, Hailey Findlay, Anderson, Lisa M, Ko, Seunghyun, Zhu, Xiang, van Deventer, Sander J, Evers, Melvin M, Konstantinova, Pavlina, Hayden, Michael R
Publicado 2020

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

por Evers, Melvin M., Miniarikova, Jana, Juhas, Stefan, Vallès, Astrid, Bohuslavova, Bozena, Juhasova, Jana, Skalnikova, Helena Kupcova, Vodicka, Petr, Valekova, Ivona, Brouwers, Cynthia, Blits, Bas, Lubelski, Jacek, Kovarova, Hana, Ellederova, Zdenka, van Deventer, Sander J., Petry, Harald, Motlik, Jan, Konstantinova, Pavlina
Publicado 2018