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Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains.

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Title: Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains.
Authors: Guo L; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA. Electronic address: lin.guo@jefferson.edu., Mann JR; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Mauna JC; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Copley KE; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wang H; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Rubien JD; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Bergmann CA; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Carey JL; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA., Merjane J; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Ngo M; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Xu J; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Odeh HM; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lin J; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lee BL; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Ganser L; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA., Robinson E; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA., Kim KM; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA., Murthy AC; Department of Molecular Biology, Cell Biology, and Biochemistry, Brown University, Providence, RI, USA., Paul T; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA., Portz B; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Gleixner AM; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Diaz Z; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Smirnov A; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Padilla G; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Lavorando E; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Espy C; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Shang Y; Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San Francisco, San Francisco, CA, USA; Department of Pathology, University of California, San Francisco, San Francisco, CA, USA., Huang EJ; Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San Francisco, San Francisco, CA, USA; Department of Pathology, University of California, San Francisco, San Francisco, CA, USA., Chesi A; Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Fawzi NL; Department of Molecular Biology, Cell Biology, and Biochemistry, Brown University, Providence, RI, USA., Myong S; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA., Donnelly CJ; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Pittsburgh Institute for Neurodegeneration, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA. Electronic address: cjdon25@pitt.edu., Shorter J; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: jshorter@pennmedicine.upenn.edu.
Source: Molecular cell [Mol Cell] 2026 Jan 08; Vol. 86 (1), pp. 114-134.e10.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 9802571 Publication Model: Print Cited Medium: Internet ISSN: 1097-4164 (Electronic) Linking ISSN: 10972765 NLM ISO Abbreviation: Mol Cell Subsets: MEDLINE
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  Data: Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains.
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  Data: <searchLink fieldCode="AU" term="%22Guo+L%22">Guo L</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA. Electronic address: lin.guo@jefferson.edu.<br /><searchLink fieldCode="AU" term="%22Mann+JR%22">Mann JR</searchLink>; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.<br /><searchLink fieldCode="AU" term="%22Mauna+JC%22">Mauna JC</searchLink>; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.<br /><searchLink fieldCode="AU" term="%22Copley+KE%22">Copley KE</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Wang+H%22">Wang H</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Rubien+JD%22">Rubien JD</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Bergmann+CA%22">Bergmann CA</searchLink>; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.<br /><searchLink fieldCode="AU" term="%22Carey+JL%22">Carey JL</searchLink>; 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Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.<br /><searchLink fieldCode="AU" term="%22Odeh+HM%22">Odeh HM</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Lin+J%22">Lin J</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Lee+BL%22">Lee BL</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Ganser+L%22">Ganser L</searchLink>; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Robinson+E%22">Robinson E</searchLink>; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Kim+KM%22">Kim KM</searchLink>; Department of Biochemistry and Molecular Biology, Thomas Jefferson University, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Murthy+AC%22">Murthy AC</searchLink>; Department of Molecular Biology, Cell Biology, and Biochemistry, Brown University, Providence, RI, USA.<br /><searchLink fieldCode="AU" term="%22Paul+T%22">Paul T</searchLink>; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Portz+B%22">Portz B</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Gleixner+AM%22">Gleixner AM</searchLink>; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.<br /><searchLink fieldCode="AU" term="%22Diaz+Z%22">Diaz Z</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Smirnov+A%22">Smirnov A</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Padilla+G%22">Padilla G</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Lavorando+E%22">Lavorando E</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Espy+C%22">Espy C</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Shang+Y%22">Shang Y</searchLink>; Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San Francisco, San Francisco, CA, USA; Department of Pathology, University of California, San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Huang+EJ%22">Huang EJ</searchLink>; Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San Francisco, San Francisco, CA, USA; Department of Pathology, University of California, San Francisco, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Chesi+A%22">Chesi A</searchLink>; Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Fawzi+NL%22">Fawzi NL</searchLink>; Department of Molecular Biology, Cell Biology, and Biochemistry, Brown University, Providence, RI, USA.<br /><searchLink fieldCode="AU" term="%22Myong+S%22">Myong S</searchLink>; Department of Biophysics, Johns Hopkins University, Baltimore, MD, USA.<br /><searchLink fieldCode="AU" term="%22Donnelly+CJ%22">Donnelly CJ</searchLink>; Department of Neurobiology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Live Like Lou Center for ALS Research, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Neuroscience, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Center for Protein Conformational Diseases, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Pittsburgh Institute for Neurodegeneration, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA. Electronic address: cjdon25@pitt.edu.<br /><searchLink fieldCode="AU" term="%22Shorter+J%22">Shorter J</searchLink>; Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: jshorter@pennmedicine.upenn.edu.
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