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

by Jolanda M. Liefhebber, Raygene Martier, Tom Van der Zon, Sonay Keskin, Angelina Huseinovic, Jacek Lubelski, Bas Blits, Harald Petry, Pavlina Konstantinova
Published 2019-06-01

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

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

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