Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.

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Title: Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Authors: Cunningham SC; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia., van Dijk EB; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia., Zhu E; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia., Sugden M; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia., Mandwie M; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia., Siew S; Department of Gastroenterology, James Fairfax Institute of Paediatric Nutrition, Sydney Children's Hospitals Network, Westmead, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia., Devanapalli B; NSW Biochemical Genetics Service, The Children's Hospital at Westmead, Westmead, Australia., Tolun AA; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; NSW Biochemical Genetics Service, The Children's Hospital at Westmead, Westmead, Australia., Klein A; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation (CSIRO), Sydney, Australia., Wilson L; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation (CSIRO), Sydney, Australia.; Department of Biomedical Sciences, Macquarie University, Macquarie Park, Australia., Aryamanesh N; Embryology Research Unit, Bioinformatics Group, Children's Medical Research Institute, University of Sydney, Westmead, Australia., Gissen P; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, United Kingdom., Baruteau J; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, University College London, London, United Kingdom., Bhattacharya K; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.; Genetic Metabolic Disorders Service, The Children's Hospital at Westmead, Sydney, Australia., Alexander IE; Gene Therapy Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney and Sydney Children's Hospitals Network, Westmead, Australia.; Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Source: Human gene therapy [Hum Gene Ther] 2023 Sep; Vol. 34 (17-18), pp. 917-926. Date of Electronic Publication: 2023 Sep 11.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1557-7422
DOI:10.1089/hum.2023.011