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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.

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Title: Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.
Authors: Jiang J; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Zhu Q; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Gendron TF; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA., Saberi S; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., McAlonis-Downes M; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Seelman A; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Stauffer JE; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Jafar-Nejad P; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Drenner K; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Schulte D; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Chun S; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Sun S; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Ling SC; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Physiology, National University of Singapore, 12 Science Drive 2, Singapore 117549, Singapore., Myers B; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Engelhardt J; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Katz M; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Baughn M; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Platoshyn O; Department of Anesthesiology, University of California, San Diego, La Jolla, CA 92093, USA; Neuroregeneration Laboratory, Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037, USA., Marsala M; Department of Anesthesiology, University of California, San Diego, La Jolla, CA 92093, USA; Neuroregeneration Laboratory, Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037, USA; Institute of Neurobiology, Slovak Academy of Sciences, Soltesovej 9, 04001 Kosice, Slovakia., Watt A; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Heyser CJ; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Ard MC; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., De Muynck L; Laboratory of Neurobiology, Vesalius Research Center, Experimental Neurology, Department of Neurosciences, KU Leuven/VIB, 3000 Leuven, Belgium; Department of Neurology, University Hospitals Leuven, 3000 Leuven, Belgium., Daughrity LM; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA., Swing DA; Mouse Cancer Genetics Program, National Cancer Institute, Frederick, MD 21702, USA., Tessarollo L; Mouse Cancer Genetics Program, National Cancer Institute, Frederick, MD 21702, USA., Jung CJ; Children's Hospital Oakland Research Institute, 5700 Martin Luther King Jr Way, Oakland, CA 94609, USA., Delpoux A; Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Molecular Biology Section, Division of Biological Sciences, University of California, San Diego, La Jolla, CA 92093, USA., Utzschneider DT; Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Molecular Biology Section, Division of Biological Sciences, University of California, San Diego, La Jolla, CA 92093, USA., Hedrick SM; Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Molecular Biology Section, Division of Biological Sciences, University of California, San Diego, La Jolla, CA 92093, USA., de Jong PJ; Children's Hospital Oakland Research Institute, 5700 Martin Luther King Jr Way, Oakland, CA 94609, USA., Edbauer D; German Center for Neurodegenerative Diseases (DZNE), Ludwig-Maximilians University and Munich Cluster of Systems Neurology (SyNergy), Feodor-Lynen Strasse 17, 81377 Munich, Germany., Van Damme P; Laboratory of Neurobiology, Vesalius Research Center, Experimental Neurology, Department of Neurosciences, KU Leuven/VIB, 3000 Leuven, Belgium; Department of Neurology, University Hospitals Leuven, 3000 Leuven, Belgium., Petrucelli L; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA., Shaw CE; Institute of Psychiatry, King's College London, London WC2R 2LS, UK., Bennett CF; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Da Cruz S; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA., Ravits J; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA., Rigo F; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA., Cleveland DW; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA. Electronic address: dcleveland@ucsd.edu., Lagier-Tourenne C; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA. Electronic address: clagier-tourenne@mgh.harvard.edu.
Source: Neuron [Neuron] 2016 May 04; Vol. 90 (3), pp. 535-50. Date of Electronic Publication: 2016 Apr 21.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 8809320 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4199 (Electronic) Linking ISSN: 08966273 NLM ISO Abbreviation: Neuron Subsets: MEDLINE
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  Data: Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.
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  Data: <searchLink fieldCode="AU" term="%22Jiang+J%22">Jiang J</searchLink>; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Zhu+Q%22">Zhu Q</searchLink>; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Gendron+TF%22">Gendron TF</searchLink>; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Saberi+S%22">Saberi S</searchLink>; Department of Neurosciences, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22McAlonis-Downes+M%22">McAlonis-Downes M</searchLink>; Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, CA 92093, USA.<br /><searchLink fieldCode="AU" term="%22Seelman+A%22">Seelman A</searchLink>; 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