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Neuron|November 1, 2024
Inhibition of RNA splicing triggers CHMP7 nuclear entry, impacting TDP-43 function and leading to the onset of ALS cellular phenotypesNorah Al-Azzam, Jenny H To, Vaishali Gautam, et al.Science Translational Medicine|October 27, 2021
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G<sub>4</sub>C<sub>2</sub>) repeat expansion in vitro and in vivo ALS modelsJessica A Bush, Haruo Aikawa, Rita Fuerst, et al.Science Translational Medicine|September 14, 2022
Poly(ADP-ribose) promotes toxicity of <i>C9ORF72</i> arginine-rich dipeptide repeat proteinsJunli Gao, Quinlan T Mewborne, Amandeep Girdhar, et al.Nature Communications|January 8, 2025
Dual-targeting CRISPR-CasRx reduces C9orf72 ALS/FTD sense and antisense repeat RNAs in vitro and in vivoLiam Kempthorne, Deniz Vaizoglu, Alexander J Cammack, et al.Nature Neuroscience|February 29, 2024
PolyGR and polyPR knock-in mice reveal a conserved neuroprotective extracellular matrix signature in C9orf72 ALS/FTD neuronsCarmelo Milioto, Mireia Carcolé, Ashling Giblin, et al.Iscience|November 8, 2021
An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients, Jonathan Li, Ryan G Lim, et al.Nature Neuroscience|February 4, 2022
Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell linesEmily G Baxi, Terri Thompson, Jonathan Li, et al.Pageof 4