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

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Bibliographic Details
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
Database: MEDLINE Ultimate
Description
ISSN:1097-4172
DOI:10.1016/j.cell.2025.07.045