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Genetic suppression features ABHD18 as a Barth syndrome therapeutic target.

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Title: Genetic suppression features ABHD18 as a Barth syndrome therapeutic target.
Authors: Masud SN; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada., Srivastava A; Scenic Biotech, Amsterdam, The Netherlands., Mero P; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Echezarreta VS; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; Program in Developmental and Stem Cell Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Anderson E; Transgenic Technology Laboratory, CRUK Scotland Institute, Glasgow, UK., van Buren L; Scenic Biotech, Amsterdam, The Netherlands., Wei J; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada., Taylor DT; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Institute for Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada., Farias AG; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada., Mikolajewicz N; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Division of Neurosurgery, Department of Surgery, University of Toronto, Toronto, Ontario, Canada., Shaw A; Institute of Human Genetics, University of Bonn, Bonn, Germany., Murareanu BM; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; McEwen Stem Cell Institute, University Health Network, Toronto, Ontario, Canada., Lohbihler M; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; McEwen Stem Cell Institute, University Health Network, Toronto, Ontario, Canada., Carney OS; Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA., van Heeringen S; Scenic Biotech, Amsterdam, The Netherlands., Clijsters L; Scenic Biotech, Amsterdam, The Netherlands., Sizova O; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., van Ameijde J; Scenic Biotech, Amsterdam, The Netherlands., Nye F; Transgenic Technology Laboratory, CRUK Scotland Institute, Glasgow, UK., Habsid A; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Nedyalkova L; Donnelly Centre, University of Toronto, Toronto, Ontario, Canada., McDonald L; Program in Developmental and Stem Cell Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Simpson C; Program in Molecular Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada., Wybenga-Groot L; Program in Molecular Medicine, The Hospital for Sick Children, Toronto, Ontario, Canada., Brown KR; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Nho N; Lundbeck La Jolla Research Center, San Diego, CA, USA., Suciu RM; Lundbeck La Jolla Research Center, San Diego, CA, USA., Chan K; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Tong AHY; Hong Kong Genome Institute, Hong Kong, China., Vaz FM; Department of Laboratory Medicine, Amsterdam UMC, Amsterdam, The Netherlands.; Core Facility Metabolomics, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands., Evers B; Scenic Biotech, Amsterdam, The Netherlands., Lesurf R; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Papaz T; Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.; Division of Cardiology, Labatt Family Heart Centre, Toronto, Ontario, Canada., Nutter LMJ; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; The Centre for Phenogenomics, The Hospital for Sick Children, Toronto, Ontario, Canada., Protze S; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; McEwen Stem Cell Institute, University Health Network, Toronto, Ontario, Canada., Billmann M; Institute of Human Genetics, University of Bonn, Bonn, Germany., Costanzo M; Donnelly Centre, University of Toronto, Toronto, Ontario, Canada., Andrews BJ; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; Donnelly Centre, University of Toronto, Toronto, Ontario, Canada., Myers CL; Department of Computer Science and Engineering, University of Minnesota, Minneapolis, MN, USA., Mital S; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Pediatrics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.; Division of Cardiology, Labatt Family Heart Centre, Toronto, Ontario, Canada.; Ted Rogers Centre for Heart Research, Toronto, Ontario, Canada., Vernon H; Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA., Brummelkamp TR; Scenic Biotech, Amsterdam, The Netherlands.; Oncode Institute, Division of Biochemistry, The Netherlands Cancer Institute, Amsterdam, The Netherlands., Boone C; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; Donnelly Centre, University of Toronto, Toronto, Ontario, Canada.; RIKEN BioResource Research Center, Kyoto, Japan., Scott IC; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.; Program in Developmental and Stem Cell Biology, The Hospital for Sick Children, Toronto, Ontario, Canada., Niphakis MJ; Lundbeck La Jolla Research Center, San Diego, CA, USA., Strathdee D; Transgenic Technology Laboratory, CRUK Scotland Institute, Glasgow, UK., Nijman SMB; Scenic Biotech, Amsterdam, The Netherlands. sebastian.nijman@proton.me., Blomen VA; Scenic Biotech, Amsterdam, The Netherlands. vincent.blomen@scenicbiotech.com., Moffat J; Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada. jason.moffat@sickkids.ca.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada. jason.moffat@sickkids.ca.; Institute for Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada. jason.moffat@sickkids.ca.
Source: Nature [Nature] 2025 Sep; Vol. 645 (8082), pp. 1029-1038. Date of Electronic Publication: 2025 Sep 03.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 0410462 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-4687 (Electronic) Linking ISSN: 00280836 NLM ISO Abbreviation: Nature Subsets: MEDLINE
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