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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
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2020.11.016