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Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS.

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Title: Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS.
Authors: McEachin ZT; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA. Electronic address: zmceach@emory.edu., Chung M; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Stratton SA; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Han C; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Kim WJ; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Sheth U; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., Thomas EV; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA., Issenberg E; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Kamra T; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Merino P; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA., Levites Y; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA., Raj N; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA., Dammer EB; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Duong DM; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Ping L; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Shantaraman A; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Trautwig AN; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Gadhavi J; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA., Assefa E; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA., Gearing M; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA., Kelly KM; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA., Roemer SF; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., DeTure M; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., Asress S; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA., Kukar T; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA., Fournier C; Department of Neurology, Emory University, Atlanta, GA 30322, USA., Dickson DW; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., Petrucelli L; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., Golde TE; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA., Oskarsson B; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA., Gendron TF; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA., Seyfried NT; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA. Electronic address: nseyfri@emory.edu., Glass JD; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA. Electronic address: jglas03@emory.edu.
Source: Cell [Cell] 2025 Nov 13; Vol. 188 (23), pp. 6424-6435.e17. Date of Electronic Publication: 2025 Aug 26.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 0413066 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4172 (Electronic) Linking ISSN: 00928674 NLM ISO Abbreviation: Cell Subsets: MEDLINE
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  Data: Molecular impact of antisense oligonucleotide therapy in C9orf72-associated ALS.
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  Data: <searchLink fieldCode="AU" term="%22McEachin+ZT%22">McEachin ZT</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA. Electronic address: zmceach@emory.edu.<br /><searchLink fieldCode="AU" term="%22Chung+M%22">Chung M</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Stratton+SA%22">Stratton SA</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Han+C%22">Han C</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Kim+WJ%22">Kim WJ</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Sheth+U%22">Sheth U</searchLink>; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Thomas+EV%22">Thomas EV</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Issenberg+E%22">Issenberg E</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Kamra+T%22">Kamra T</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Merino+P%22">Merino P</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Levites+Y%22">Levites Y</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Raj+N%22">Raj N</searchLink>; Department of Human Genetics, Emory University, Atlanta, GA 30322, USA; Department of Cell Biology, Emory University, Atlanta, GA 30322, USA; Laboratory for Translational Cell Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Dammer+EB%22">Dammer EB</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Duong+DM%22">Duong DM</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Ping+L%22">Ping L</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Shantaraman+A%22">Shantaraman A</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Trautwig+AN%22">Trautwig AN</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Gadhavi+J%22">Gadhavi J</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Assefa+E%22">Assefa E</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Gearing+M%22">Gearing M</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Kelly+KM%22">Kelly KM</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Roemer+SF%22">Roemer SF</searchLink>; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22DeTure+M%22">DeTure M</searchLink>; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Asress+S%22">Asress S</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Kukar+T%22">Kukar T</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Fournier+C%22">Fournier C</searchLink>; Department of Neurology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Dickson+DW%22">Dickson DW</searchLink>; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Petrucelli+L%22">Petrucelli L</searchLink>; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Golde+TE%22">Golde TE</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pharmacology & Chemical Biology, Emory University, Atlanta, GA 30322, USA.<br /><searchLink fieldCode="AU" term="%22Oskarsson+B%22">Oskarsson B</searchLink>; Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Gendron+TF%22">Gendron TF</searchLink>; Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Jacksonville, FL 32224, USA; Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.<br /><searchLink fieldCode="AU" term="%22Seyfried+NT%22">Seyfried NT</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Biochemistry, Emory University, Atlanta, GA 30322, USA. Electronic address: nseyfri@emory.edu.<br /><searchLink fieldCode="AU" term="%22Glass+JD%22">Glass JD</searchLink>; Goizueta Brain Health Institute Center for Neurodegenerative Diseases, Emory University, Atlanta, GA 30322, USA; Department of Neurology, Emory University, Atlanta, GA 30322, USA; Department of Pathology, Emory University, Atlanta, GA 30322, USA. Electronic address: jglas03@emory.edu.
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