Martier R, Liefhebber JM, García-Osta A, Miniarikova J, Cuadrado-Tejedor M, Espelosin M, Ursua S, Petry H, van Deventer SJ, Evers MM, et al. Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy. Mol Ther Nucleic Acids. 2019;16:26-37.