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The EMBO Journal|May 30, 2023
HNRNPH1 regulates the neuroprotective cold-shock protein RBM3 expression through poison exon exclusionJulie Qiaojin Lin, Deepak Khuperkar, Sofia Pavlou, et al.Brain, Behavior, and Immunity|September 16, 2023
P2X<sub>7</sub>R influences tau aggregate burden in human tauopathies and shows distinct signalling in microglia and astrocytesPaula Beltran-Lobo, Martina M Hughes, Claire Troakes, et al.Cell Chemical Biology|March 23, 2021
Repurposing of glycine transport inhibitors for the treatment of erythropoietic protoporphyriaFrançois Halloy, Pavithra S Iyer, Alice Ghidini, et al.Nature Communications|June 25, 2026
Karyoptosis mediates cell death and neurodegeneration upon proteotoxic stressRebecca Casterton, Aitana Martinez-Cotrina, Jodi Barnard, et al.Acta Neuropathologica|February 20, 2016
Monomethylated and unmethylated FUS exhibit increased binding to Transportin and distinguish FTLD-FUS from ALS-FUSMarc Suárez-Calvet, Manuela Neumann, Thomas Arzberger, et al.Biorxiv : the Preprint Server for Biology|February 27, 2026
Breaking β-sheets in FUS prion-like domain preserves phase separation and function but prevents aggregation and toxicityNoah Wake, Juan Alcalde, Daniel Jutzi, et al.Nature Communications|December 8, 2019
Muscleblind acts as a modifier of FUS toxicity by modulating stress granule dynamics and SMN localizationIan Casci, Karthik Krishnamurthy, Sukhleen Kour, et al.Cellular and Molecular Life Sciences : CMLS|November 25, 2023
Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2AFederica Rizzo, Silvia Bono, Marc David Ruepp, et al.Biorxiv : the Preprint Server for Biology|October 1, 2025
Generation of <i>C9orf72</i> repeat knock-in iPSC lines for modelling ALS and FTDRachel Coneys, Alexander J Cammack, Remya R Nair, et al.Biorxiv : the Preprint Server for Biology|December 15, 2025
U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral SclerosisPuja R Mehta, Tomas Solomon, Sarah Pickles, et al.Pageof 6