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

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

Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing

by Karine Cambon, Virginie Zimmer, Sylvain Martineau, Marie-Claude Gaillard, Margot Jarrige, Aurore Bugi, Jana Miniarikova, Maria Rey, Raymonde Hassig, Noelle Dufour, Gwenaelle Auregan, Philippe Hantraye, Anselme L. Perrier, Nicole Déglon
Published 2017-06-01