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- C9ORF72 poly(GA) aggregates sequester and impair HR23 and nucleocytoplasmic transport proteins Journal Article
- ALS/FTD mutations in UBQLN2 impede autophagy by reducing autophagosome acidification through loss of function Journal Article
- Allele-Selective Thiomorpholino Antisense Oligonucleotides as a Therapeutic Approach for Fused-in-Sarcoma Amyotrophic Lateral Sclerosis Journal Article
- C9orf72 and triplet repeat disorder RNAs: G-quadruplex formation, binding to PRC2 and implications for disease mechanisms Journal Article
- Distinct brain transcriptome profiles in C9orf72-associated and sporadic ALS Journal Article ... more