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

por Liefhebber, Jolanda M., Martier, Raygene, Van der Zon, Tom, Keskin, Sonay, Huseinovic, Angelina, Lubelski, Jacek, Blits, Bas, Petry, Harald, Konstantinova, Pavlina
Publicado 2019

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

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

Proof-of-concept study for liver-directed miQURE technology in a dyslipidemic mouse model

por Zancanella, Vanessa, Vallès, Astrid, Liefhebber, Jolanda M.P., Paerels, Lieke, Tornero, Carlos Vendrell, Wattimury, Hendrina, van der Zon, Tom, van Rooijen, Kristel, Golinska, Monika, Grevelink, Tamar, Ehlert, Erich, Pieterman, Elsbet Jantine, Keijzer, Nanda, Princen, Hans Marinus Gerardus, Stokman, Geurt, Liu, Ying Poi
Publicado 2023