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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.

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Title: Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
Authors: De Sabbata G; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy., Boisgerault F; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Guarnaccia C; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy., Iaconcig A; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy., Bortolussi G; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy., Collaud F; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Ronzitti G; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Sola MS; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Vidal P; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Rouillon J; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Charles S; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France., Nicastro E; Ospedale Papa Giovanni XXIII, 24127 Bergamo, BG, Italy., D'Antiga L; Ospedale Papa Giovanni XXIII, 24127 Bergamo, BG, Italy., Ilyinskii P; Selecta Biosciences, Watertown, MA 02472, USA., Mingozzi F; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.; Institut de Myologie, 73013 Paris, France., Kishimoto TK; Selecta Biosciences, Watertown, MA 02472, USA., Muro AF; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Nov 17; Vol. 20, pp. 169-180. Date of Electronic Publication: 2020 Nov 17 (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.2020.11.005