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RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.
Donnelly CJ, Zhang PW, Pham JT, Haeusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD. Donnelly CJ, et al. Among authors: wang j. Neuron. 2013 Oct 16;80(2):415-28. doi: 10.1016/j.neuron.2013.10.015. Neuron. 2013. PMID: 24139042 Free PMC article.
C9orf72 nucleotide repeat structures initiate molecular cascades of disease.
Haeusler AR, Donnelly CJ, Periz G, Simko EA, Shaw PG, Kim MS, Maragakis NJ, Troncoso JC, Pandey A, Sattler R, Rothstein JD, Wang J. Haeusler AR, et al. Among authors: wang j. Nature. 2014 Mar 13;507(7491):195-200. doi: 10.1038/nature13124. Epub 2014 Mar 5. Nature. 2014. PMID: 24598541 Free PMC article.
The C9orf72 repeat expansion disrupts nucleocytoplasmic transport.
Zhang K, Donnelly CJ, Haeusler AR, Grima JC, Machamer JB, Steinwald P, Daley EL, Miller SJ, Cunningham KM, Vidensky S, Gupta S, Thomas MA, Hong I, Chiu SL, Huganir RL, Ostrow LW, Matunis MJ, Wang J, Sattler R, Lloyd TE, Rothstein JD. Zhang K, et al. Among authors: wang j. Nature. 2015 Sep 3;525(7567):56-61. doi: 10.1038/nature14973. Epub 2015 Aug 26. Nature. 2015. PMID: 26308891 Free PMC article.
A zebrafish model for C9orf72 ALS reveals RNA toxicity as a pathogenic mechanism.
Swinnen B, Bento-Abreu A, Gendron TF, Boeynaems S, Bogaert E, Nuyts R, Timmers M, Scheveneels W, Hersmus N, Wang J, Mizielinska S, Isaacs AM, Petrucelli L, Lemmens R, Van Damme P, Van Den Bosch L, Robberecht W. Swinnen B, et al. Among authors: wang j. Acta Neuropathol. 2018 Mar;135(3):427-443. doi: 10.1007/s00401-017-1796-5. Epub 2018 Jan 4. Acta Neuropathol. 2018. PMID: 29302778
Autophagy as a common pathway in amyotrophic lateral sclerosis.
Nguyen DKH, Thombre R, Wang J. Nguyen DKH, et al. Among authors: wang j. Neurosci Lett. 2019 Apr 1;697:34-48. doi: 10.1016/j.neulet.2018.04.006. Epub 2018 Apr 4. Neurosci Lett. 2019. PMID: 29626651 Free PMC article. Review.
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