Valentina Sardone

Hexanucleotide Repeats in ALS/FTD Form Length-Dependent RNA Foci, Sequester RNA Binding Proteins, and Are Neurotoxic

Youn‐Bok Lee, Han-Jou Chen, João Peres et al. · Cell Reports · 2013 · 483 citations · Full text

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

Diane E. Frank, Frederick J. Schnell, Cody Akana et al. · Neurology · 2020 · 310 citations · Full text

Differential roles of the ubiquitin proteasome system (UPS) and autophagy in the clearance of soluble and aggregated TDP-43 species

Emma L. Scotter, Caroline Vance, Agnes L. Nishimura et al. · Journal of Cell Science · 2014 · 266 citations · Full text

Retention of hexanucleotide repeat-containing intron in C9orf72 mRNA: implications for the pathogenesis of ALS/FTD

Michael Niblock, Bradley Smith, Youn‐Bok Lee et al. · Acta Neuropathologica Communications · 2016 · 65 citations · Full text

Allele-Specific Knockdown of ALS-Associated Mutant TDP-43 in Neural Stem Cells Derived from Induced Pluripotent Stem Cells

Agnes L. Nishimura, Carole Shum, Emma L. Scotter et al. · PLoS ONE · 2014 · 48 citations · Full text