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Functional editing of the OTC locus by targeted integration with phenotype correction and restoration of endogenous expression patterns.

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Title: Functional editing of the OTC locus by targeted integration with phenotype correction and restoration of endogenous expression patterns.
Authors: Ginn SL; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia. Electronic address: sginn@cmri.org.au., Doroudian F; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Christina S; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Chan OPY; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Lucas CW; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Zhu E; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Yang SF; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Devanapalli B; NSW Biochemical Genetics Service, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia., Klein AH; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation (CSIRO), Herston, QLD 4006, Australia., Scott S; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Vitale J; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Cunningham SC; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Liao SHY; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Cabanes-Creus M; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia., Lisowski L; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine, National Research Institute, 04-141 Warszawa, Poland., Alexander IE; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jul 09. Date of Electronic Publication: 2026 Jul 09.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Functional editing of the OTC locus by targeted integration with phenotype correction and restoration of endogenous expression patterns.
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  Data: <searchLink fieldCode="AU" term="%22Ginn+SL%22">Ginn SL</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia. Electronic address: sginn@cmri.org.au.<br /><searchLink fieldCode="AU" term="%22Doroudian+F%22">Doroudian F</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Christina+S%22">Christina S</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Chan+OPY%22">Chan OPY</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Lucas+CW%22">Lucas CW</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Zhu+E%22">Zhu E</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Yang+SF%22">Yang SF</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Devanapalli+B%22">Devanapalli B</searchLink>; NSW Biochemical Genetics Service, The Children's Hospital at Westmead, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Klein+AH%22">Klein AH</searchLink>; Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation (CSIRO), Herston, QLD 4006, Australia.<br /><searchLink fieldCode="AU" term="%22Scott+S%22">Scott S</searchLink>; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Vitale+J%22">Vitale J</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Cunningham+SC%22">Cunningham SC</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Liao+SHY%22">Liao SHY</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Cabanes-Creus+M%22">Cabanes-Creus M</searchLink>; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.<br /><searchLink fieldCode="AU" term="%22Lisowski+L%22">Lisowski L</searchLink>; Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine, National Research Institute, 04-141 Warszawa, Poland.<br /><searchLink fieldCode="AU" term="%22Alexander+IE%22">Alexander IE</searchLink>; Gene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia; Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2026 Jul 09. <i>Date of Electronic Publication: </i>2026 Jul 09.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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      – Type: doi
        Value: 10.1016/j.ymthe.2026.06.044
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      – Code: eng
        Text: English
    Titles:
      – TitleFull: Functional editing of the OTC locus by targeted integration with phenotype correction and restoration of endogenous expression patterns.
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            – D: 09
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              Text: 2026 Jul 09
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              Y: 2026
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