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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.
Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C. Jiang J, et al. Among authors: sun s. Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21. Neuron. 2016. PMID: 27112497 Free PMC article.
CRISPR-Cas9 Screens Identify the RNA Helicase DDX3X as a Repressor of C9ORF72 (GGGGCC)n Repeat-Associated Non-AUG Translation.
Cheng W, Wang S, Zhang Z, Morgens DW, Hayes LR, Lee S, Portz B, Xie Y, Nguyen BV, Haney MS, Yan S, Dong D, Coyne AN, Yang J, Xian F, Cleveland DW, Qiu Z, Rothstein JD, Shorter J, Gao FB, Bassik MC, Sun S. Cheng W, et al. Among authors: sun s. Neuron. 2019 Dec 4;104(5):885-898.e8. doi: 10.1016/j.neuron.2019.09.003. Epub 2019 Oct 3. Neuron. 2019. PMID: 31587919 Free PMC article.
Elevated nuclear TDP-43 induces constitutive exon skipping.
Carmen-Orozco RP, Tsao W, Ye Y, Sinha IR, Chang K, Trinh V, Chung W, Bowden K, Troncoso JC, Blackshaw S, Hayes LR, Sun S, Wong PC, Ling JP. Carmen-Orozco RP, et al. Among authors: sun s. bioRxiv [Preprint]. 2023 May 12:2023.05.11.540291. doi: 10.1101/2023.05.11.540291. bioRxiv. 2023. Update in: Mol Neurodegener. 2024 Jun 9;19(1):45. doi: 10.1186/s13024-024-00732-w PMID: 37215013 Free PMC article. Updated. Preprint.
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