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Rescue of glutaric aciduria type I in mice by liver-directed therapies.

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Title: Rescue of glutaric aciduria type I in mice by liver-directed therapies.
Authors: Barzi M; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Johnson CG; Center for Cell and Gene Therapy, Stem Cells and Regenerative Medicine Center, Baylor College of Medicine, Houston, TX 77030, USA., Chen T; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Rodriguiz RM; Department of Psychiatry and Behavioral Sciences, Cell Biology and Neurobiology, Mouse Behavioral and Neuroendocrine Analysis Core Facility, Duke University Medical Center, Durham, NC 27710, USA., Hemmingsen M; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Gonzalez TJ; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA., Rosales A; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA., Beasley J; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Peck CK; Biochemical Genetics Laboratory, Children's Hospital Colorado Anschutz Medical Campus, Aurora, CO 80045, USA., Ma Y; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Stiles AR; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Wood TC; Biochemical Genetics Laboratory, Children's Hospital Colorado Anschutz Medical Campus, Aurora, CO 80045, USA., Maeso-Diaz R; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA., Diehl AM; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA., Young SP; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Everitt JI; Department of Pathology, Duke University Medical Center, Durham, NC 27710, USA., Wetsel WC; Department of Psychiatry and Behavioral Sciences, Cell Biology and Neurobiology, Mouse Behavioral and Neuroendocrine Analysis Core Facility, Duke University Medical Center, Durham, NC 27710, USA., Lagor WR; Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA., Bissig-Choisat B; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Asokan A; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA.; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.; Department of Biomedical Engineering (BME) at the Duke University Pratt School of Engineering, Duke University Medical Center, Durham, NC 27710, USA.; Duke Cancer Center, Duke University Medical Center, Durham, NC 27710, USA., El-Gharbawy A; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA., Bissig KD; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA.; Department of Biomedical Engineering (BME) at the Duke University Pratt School of Engineering, Duke University Medical Center, Durham, NC 27710, USA.; Duke Cancer Center, Duke University Medical Center, Durham, NC 27710, USA.; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, NC 27710, USA.
Source: Science translational medicine [Sci Transl Med] 2023 Apr 19; Vol. 15 (692), pp. eadf4086. Date of Electronic Publication: 2023 Apr 19.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Rescue of glutaric aciduria type I in mice by liver-directed therapies.
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  Data: <searchLink fieldCode="AU" term="%22Barzi+M%22">Barzi M</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+CG%22">Johnson CG</searchLink>; Center for Cell and Gene Therapy, Stem Cells and Regenerative Medicine Center, Baylor College of Medicine, Houston, TX 77030, USA.<br /><searchLink fieldCode="AU" term="%22Chen+T%22">Chen T</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Rodriguiz+RM%22">Rodriguiz RM</searchLink>; Department of Psychiatry and Behavioral Sciences, Cell Biology and Neurobiology, Mouse Behavioral and Neuroendocrine Analysis Core Facility, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Hemmingsen+M%22">Hemmingsen M</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Gonzalez+TJ%22">Gonzalez TJ</searchLink>; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Rosales+A%22">Rosales A</searchLink>; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Beasley+J%22">Beasley J</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Peck+CK%22">Peck CK</searchLink>; Biochemical Genetics Laboratory, Children's Hospital Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.<br /><searchLink fieldCode="AU" term="%22Ma+Y%22">Ma Y</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Stiles+AR%22">Stiles AR</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Wood+TC%22">Wood TC</searchLink>; Biochemical Genetics Laboratory, Children's Hospital Colorado Anschutz Medical Campus, Aurora, CO 80045, USA.<br /><searchLink fieldCode="AU" term="%22Maeso-Diaz+R%22">Maeso-Diaz R</searchLink>; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Diehl+AM%22">Diehl AM</searchLink>; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Young+SP%22">Young SP</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Everitt+JI%22">Everitt JI</searchLink>; Department of Pathology, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Wetsel+WC%22">Wetsel WC</searchLink>; Department of Psychiatry and Behavioral Sciences, Cell Biology and Neurobiology, Mouse Behavioral and Neuroendocrine Analysis Core Facility, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Lagor+WR%22">Lagor WR</searchLink>; Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.<br /><searchLink fieldCode="AU" term="%22Bissig-Choisat+B%22">Bissig-Choisat B</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Asokan+A%22">Asokan A</searchLink>; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27710, USA.; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.; Department of Biomedical Engineering (BME) at the Duke University Pratt School of Engineering, Duke University Medical Center, Durham, NC 27710, USA.; Duke Cancer Center, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22El-Gharbawy+A%22">El-Gharbawy A</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.<br /><searchLink fieldCode="AU" term="%22Bissig+KD%22">Bissig KD</searchLink>; Y.T. and Alice Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.; Department of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC 27710, USA.; Department of Biomedical Engineering (BME) at the Duke University Pratt School of Engineering, Duke University Medical Center, Durham, NC 27710, USA.; Duke Cancer Center, Duke University Medical Center, Durham, NC 27710, USA.; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, NC 27710, USA.
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  Data: <searchLink fieldCode="JN" term="%22101505086%22">Science translational medicine</searchLink> [Sci Transl Med] 2023 Apr 19; Vol. 15 (692), pp. eadf4086. <i>Date of Electronic Publication: </i>2023 Apr 19.
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