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

by Kimberly L. Pietersz, Kimberly L. Pietersz, Francois Du Plessis, Stephan M. Pouw, Jolanda M. Liefhebber, Sander J. van Deventer, Sander J. van Deventer, Gerard J. M. Martens, Pavlina S. Konstantinova, Bas Blits
Published 2021-08-01

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

by Elisabeth A. Spronck, Cynthia C. Brouwers, Astrid Vallès, Martin de Haan, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Published 2019-06-01

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

by Sonay Keskin, Cynthia C. Brouwers, Marina Sogorb-Gonzalez, Raygene Martier, Josse A. Depla, Astrid Vallès, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Published 2019-12-01

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

by 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
Published 2019-03-01

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

by 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
Published 2020-09-01

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

by 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
Published 2019-06-01

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

by 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
Published 2019-12-01

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

by 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
Published 2021-01-01

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

by 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
Published 2016-01-01