Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.

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Title: Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Authors: Shi X; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Aronson SJ; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Ten Bloemendaal L; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Duijst S; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Bakker RS; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., de Waart DR; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Bortolussi G; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Collaud F; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, INSERM, Genethon, Integrare Research Unit UMR S951, 91000 Evry, France., Oude Elferink RP; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Muro AF; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Mingozzi F; Genethon, 91000 Evry, France., Ronzitti G; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, INSERM, Genethon, Integrare Research Unit UMR S951, 91000 Evry, France., Bosma PJ; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Dec 03; Vol. 20, pp. 287-297. Date of Electronic Publication: 2020 Dec 03 (Print Publication: 2021).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Shi+X%22">Shi X</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Aronson+SJ%22">Aronson SJ</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Ten+Bloemendaal+L%22">Ten Bloemendaal L</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Duijst+S%22">Duijst S</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Bakker+RS%22">Bakker RS</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22de+Waart+DR%22">de Waart DR</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Bortolussi+G%22">Bortolussi G</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, INSERM, Genethon, Integrare Research Unit UMR S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Oude+Elferink+RP%22">Oude Elferink RP</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.<br /><searchLink fieldCode="AU" term="%22Muro+AF%22">Muro AF</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; Genethon, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, INSERM, Genethon, Integrare Research Unit UMR S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Bosma+PJ%22">Bosma PJ</searchLink>; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AGEM, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.
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