Tom van der Zon - UniQure NV, Vector Technol, Amsterdam, Netherlands

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Proof-of-concept Study for Liver-Directed Miqure Technology in a Dyslipidemic Mouse Model

Vanessa Zancanella,Astrid Vallès,Jolanda M.P. Liefhebber,Lieke Paerels, Carlos Vendrell Tornero,Hendrina Wattimury,Tom van der Zon,Kristel van Rooijen,Monika Golinska, Tamar Grevelink,Erich Ehlert,Elsbet J. Pieterman,Nanda Keijzer,Hans Marinus Gerardus Princen,Geurt Stokman,Ying Poi Liu

Molecular therapy Nucleic acidsno. 2 (2024): 102189-102189

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Proof-of-concept Study for Liver-Directed Miqure Technology in a Dyslipidemic Mouse Model

Vanessa Zancanella,Astrid Valles,Jolanda M. P. Liefhebber,Lieke Paerels, Carlos Vendrell Tornero,Hendrina Wattimury,Tom van der Zon,Kristel van Rooijen,Monika Golinska, Tamar Grevelink,Erich Ehlert,Elsbet Jantine Pieterman,Nanda Keijzer,Hans Marinus Gerardus Princen,Geurt Stokman,Ying Poi Liu

Molecular Therapy - Nucleic Acids (2023): 454-467

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Novel Method of Producing AAV Capsid Libraries in Insect Cells

Bas Bosma, Elena Santidrian Yebra-Pimentel,Tom van der Zon, Lyubomir Momchev,Milja Pesic,Noun Bashir, Marsha Haneveld,Angga Kusuma

MOLECULAR THERAPY（2023）

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The Capsid Race: Multiplexed Analysis and Comparison of Rationally Engineered AAV CNS Capsids In Vivo

Elena Pimentel, Sebastian Kieper,Fiona Moreno,Tom Van der Zon,Bas Bosma,Noun Bashir,Stijn Waaijer, Pedro Espinosa, Tycho Hoogland,Mercedes Valls,Milja Pesic,Angga Kusuma

MOLECULAR THERAPY（2023）

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Selective Silencing of Mutant C9orf72 Transcripts

Vanessa Zancanella, Sebastian Kieper,Irena Bockaj, Maroeska Oudshoorn-Dickmann,Kristel van Rooijen, Vlad Shtefaniuk,Tom van der Zon, Bianca Zardetto,Mercedes Valls, Leontien van der Bent,Morgane Wartel,Ying Poi Liu

MOLECULAR THERAPY（2022）

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Development of an AAV5-based gene therapy for Fabry disease

Ying Poi Liu,Jolanda M.P. Liefhebber,Tom van der Zon,Lieke Paerels,Maria J. Ferraz,Roelof Ottenhoff,Vanessa Zancanella, Betty Au,Chi-Lin Kuo,Carlie J.M. de Vries,Melvin M. Evers,Pavlina Konstantinova, Johannes M.F.G. Aerts,Sander J. Van Deventer

MOLECULAR GENETICS AND METABOLISM（2020）

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In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver

Jolanda M Liefhebber,Raygene Martier,Tom Van der Zon,Sonay Keskin,Angelina Huseinovic,Jacek Lubelski,Bas Blits,Harald Petry,Pavlina Konstantinova

Molecular therapy. Methods & clinical development（2019）

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

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

Molecular therapy Nucleic acids（2019）

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

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

Molecular therapy Nucleic acids（2016）

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