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Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

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Title: Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
Authors: Shi X; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.; School of Life Sciences, Liaoning Provincial Key Laboratory of Biotechnology and Drug Discovery, Liaoning Normal University, Dalian 116082, P.R. China., Bortolussi G; International Centre for Genetic Engineering and Biotechnology (ICGEB), Padriciano 99, 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., Le Brun PR; Genethon, 91000 Evry, France., Bloemendaal LT; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Guerchet N; Genethon, 91000 Evry, France., Rudi de Waart D; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Sellier P; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Duijst S; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Veron P; Genethon, 91000 Evry, France., Mingozzi F; Genethon, 91000 Evry, France., Kishimoto TK; Selecta Biosciences, Watertown, MA 02472, USA., Ronzitti G; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Bosma P; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Muro AF; International Centre for Genetic Engineering and Biotechnology (ICGEB), Padriciano 99, 34149 Trieste, Italy.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Oct 28; Vol. 32 (4), pp. 101363. Date of Electronic Publication: 2024 Oct 28 (Print Publication: 2024).
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.2024.101363