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Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases.

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Title: Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases.
Authors: Gurung S; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK., Perocheau D; Great Ormond Street Institute of Child Health, University College London, London, UK., Ghosh R; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK., Hart SL; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK., Baruteau J; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK.; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2025 Sep; Vol. 48 (5), pp. e70078.
Publication Type: Journal Article; Review
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Gurung+S%22">Gurung S</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Perocheau+D%22">Perocheau D</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Ghosh+R%22">Ghosh R</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Hart+SL%22">Hart SL</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Baruteau+J%22">Baruteau J</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.; National Institute of Health Research Great Ormond Street Biomedical Research Centre, London, UK.; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
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        Value: 10.1002/jimd.70078
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              Text: 2025 Sep
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