AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model
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
Published in Molecular therapy (05.09.2018)
Published in Molecular therapy (05.09.2018)
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Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy
Ferreira, Valerie, Twisk, Jaap, Kwikkers, Karin, Aronica, Eleonora, Brisson, Diane, Methot, Julie, Petry, Harald, Gaudet, Daniel
Published in Human gene therapy (01.03.2014)
Published in Human gene therapy (01.03.2014)
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A largely random AAV integration profile after LPLD gene therapy
Kaeppel, Christine, Beattie, Stuart G, Fronza, Raffaele, van Logtenstein, Richard, Salmon, Florence, Schmidt, Sabine, Wolf, Stephan, Nowrouzi, Ali, Glimm, Hanno, von Kalle, Christof, Petry, Harald, Gaudet, Daniel, Schmidt, Manfred
Published in Nature medicine (01.07.2013)
Published in Nature medicine (01.07.2013)
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Effect of Alipogene Tiparvovec (AAV1-LPLS447X) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients
Carpentier, André C, Frisch, Frédérique, Labbé, Sébastien M, Gagnon, René, de Wal, Janneke, Greentree, Stephen, Petry, Harald, Twisk, Jaap, Brisson, Diane, Gaudet, Daniel
Published in The journal of clinical endocrinology and metabolism (01.05.2012)
Published in The journal of clinical endocrinology and metabolism (01.05.2012)
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Adenosine triphosphate-binding cassette transporter genes up-regulation in untreated hepatocellular carcinoma is mediated by cellular microRNAs
Borel, Florie, Han, Ruiqi, Visser, Allerdien, Petry, Harald, van Deventer, Sander J.H., Jansen, Peter L.M., Konstantinova, Pavlina
Published in Hepatology (Baltimore, Md.) (01.03.2012)
Published in Hepatology (Baltimore, Md.) (01.03.2012)
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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy
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
Published in Molecular therapy. Nucleic acids (07.06.2019)
Published in Molecular therapy. Nucleic acids (07.06.2019)
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Journal Article
Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients
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
Published in Molecular therapy. Nucleic acids (01.03.2019)
Published in Molecular therapy. Nucleic acids (01.03.2019)
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Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease
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
Published in Molecular therapy. Nucleic acids (2016)
Published in Molecular therapy. Nucleic acids (2016)
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Molecular therapy for obesity and diabetes based on a long‐term increase in hepatic fatty‐acid oxidation
Orellana‐Gavaldà, Josep M., Herrero, Laura, Malandrino, Maria Ida, Pañeda, Astrid, Sol Rodríguez‐Peña, Maria, Petry, Harald, Asins, Guillermina, Van Deventer, Sander, Hegardt, Fausto G., Serra, Dolors
Published in Hepatology (Baltimore, Md.) (01.03.2011)
Published in Hepatology (Baltimore, Md.) (01.03.2011)
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Journal Article
AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models
Spronck, Elisabeth A., Brouwers, Cynthia C., Vallès, Astrid, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Konstantinova, Pavlina, Evers, Melvin M.
Published in Molecular therapy. Methods & clinical development (14.06.2019)
Published in Molecular therapy. Methods & clinical development (14.06.2019)
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Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates
Salas, David, Kwikkers, Karin L., Zabaleta, Nerea, Bazo, Andrea, Petry, Harald, van Deventer, Sander J., Aseguinolaza, Gloria Gonzalez, Ferreira, Valerie
Published in Blood advances (10.09.2019)
Published in Blood advances (10.09.2019)
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Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates
Unzu, Carmen, Hervás-Stubbs, Sandra, Sampedro, Ana, Mauleón, Itsaso, Mancheño, Uxua, Alfaro, Carlos, de Salamanca, Rafael Enríquez, Benito, Alberto, Beattie, Stuart G, Petry, Harald, Prieto, Jesús, Melero, Ignacio, Fontanellas, Antonio
Published in Journal of translational medicine (15.06.2012)
Published in Journal of translational medicine (15.06.2012)
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Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5 ch and AAV1
Majowicz, Anna, Salas, David, Zabaleta, Nerea, Rodríguez-Garcia, Estefania, González-Aseguinolaza, Gloria, Petry, Harald, Ferreira, Valerie
Published in Molecular therapy (02.08.2017)
Published in Molecular therapy (02.08.2017)
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Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs
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.
Published in Molecular therapy. Methods & clinical development (13.12.2019)
Published in Molecular therapy. Methods & clinical development (13.12.2019)
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Journal Article
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
Spronck, Elisabeth A, Vallès, Astrid, Lampen, Margit H, Montenegro-Miranda, Paula S, Keskin, Sonay, Heijink, Liesbeth, Evers, Melvin M, Petry, Harald, Deventer, Sander J van, Konstantinova, Pavlina, Haan, Martin de
Published in Brain sciences (20.01.2021)
Published in Brain sciences (20.01.2021)
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Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice
Unzu, Carmen, Sampedro, Ana, Mauleón, Itsaso, Alegre, Manuel, Beattie, Stuart G, de Salamanca, Rafael Enríquez, Snapper, Jolanda, Twisk, Jaap, Petry, Harald, González-Aseguinolaza, Gloria, Artieda, Julio, Rodríguez-Pena, María Sol, Prieto, Jesús, Fontanellas, Antonio
Published in Molecular therapy (01.02.2011)
Published in Molecular therapy (01.02.2011)
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Long-term increased carnitine palmitoyltransferase 1A expression in ventromedial hypotalamus causes hyperphagia and alters the hypothalamic lipidomic profile
Mera, Paula, Mir, Joan Francesc, Fabriàs, Gemma, Casas, Josefina, Costa, Ana S H, Malandrino, Maria Ida, Fernández-López, José-Antonio, Remesar, Xavier, Gao, Su, Chohnan, Shigeru, Rodríguez-Peña, Maria Sol, Petry, Harald, Asins, Guillermina, Hegardt, Fausto G, Herrero, Laura, Serra, Dolors
Published in PloS one (12.05.2014)
Published in PloS one (12.05.2014)
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In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver
Liefhebber, Jolanda M., Martier, Raygene, Van der Zon, Tom, Keskin, Sonay, Huseinovic, Angelina, Lubelski, Jacek, Blits, Bas, Petry, Harald, Konstantinova, Pavlina
Published in Molecular therapy. Methods & clinical development (14.06.2019)
Published in Molecular therapy. Methods & clinical development (14.06.2019)
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Optimization and comparison of knockdown efficacy between polymerase II expressed shRNA and artificial miRNA targeting luciferase and Apolipoprotein B100
Maczuga, Piotr, Koornneef, Annemart, Borel, Florie, Petry, Harald, van Deventer, Sander, Ritsema, Tita, Konstantinova, Pavlina
Published in BMC biotechnology (24.07.2012)
Published in BMC biotechnology (24.07.2012)
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