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Rescue of glutaric aciduria type I in mice by liver-directed therapies.
Barzi, Mercedes; Johnson, Collin G; Chen, Tong; Rodriguiz, Ramona M; Hemmingsen, Madeline; Gonzalez, Trevor J; Rosales, Alan; Beasley, James; Peck, Cheryl K; Ma, Yunhan; Stiles, Ashlee R; Wood, Timothy C; Maeso-Diaz, Raquel; Diehl, Anna Mae; Young, Sarah P; Everitt, Jeffrey I; Wetsel, William C; Lagor, William R; Bissig-Choisat, Beatrice; Asokan, Aravind; El-Gharbawy, Areeg; Bissig, Karl-Dimiter.
Affiliation
  • 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.
  • El-Gharbawy A; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.
  • Bissig KD; Department of Biomedical Engineering (BME) at the Duke University Pratt School of Engineering, Duke University Medical Center, Durham, NC 27710, USA.
Sci Transl Med ; 15(692): eadf4086, 2023 04 19.
Article in En | MEDLINE | ID: mdl-37075130
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glutaryl-CoA Dehydrogenase / Lysine Type of study: Prognostic_studies Limits: Animals Language: En Journal: Sci Transl Med Journal subject: CIENCIA / MEDICINA Year: 2023 Document type: Article Affiliation country: Estados Unidos
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glutaryl-CoA Dehydrogenase / Lysine Type of study: Prognostic_studies Limits: Animals Language: En Journal: Sci Transl Med Journal subject: CIENCIA / MEDICINA Year: 2023 Document type: Article Affiliation country: Estados Unidos