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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
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  Data: Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
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  Data: <searchLink fieldCode="AU" term="%22De+Sabbata+G%22">De Sabbata G</searchLink>; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Boisgerault+F%22">Boisgerault F</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Guarnaccia+C%22">Guarnaccia C</searchLink>; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Iaconcig+A%22">Iaconcig A</searchLink>; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Bortolussi+G%22">Bortolussi G</searchLink>; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Sola+MS%22">Sola MS</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Vidal+P%22">Vidal P</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Rouillon+J%22">Rouillon J</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Charles+S%22">Charles S</searchLink>; Généthon, 91000 Evry, France.; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.<br /><searchLink fieldCode="AU" term="%22Nicastro+E%22">Nicastro E</searchLink>; Ospedale Papa Giovanni XXIII, 24127 Bergamo, BG, Italy.<br /><searchLink fieldCode="AU" term="%22D'Antiga+L%22">D'Antiga L</searchLink>; Ospedale Papa Giovanni XXIII, 24127 Bergamo, BG, Italy.<br /><searchLink fieldCode="AU" term="%22Ilyinskii+P%22">Ilyinskii P</searchLink>; Selecta Biosciences, Watertown, MA 02472, USA.<br /><searchLink fieldCode="AU" term="%22Mingozzi+F%22">Mingozzi F</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Kishimoto+TK%22">Kishimoto TK</searchLink>; Selecta Biosciences, Watertown, MA 02472, USA.<br /><searchLink fieldCode="AU" term="%22Muro+AF%22">Muro AF</searchLink>; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
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  Data: <searchLink fieldCode="JN" term="%22101624857%22">Molecular therapy. Methods & clinical development</searchLink> [Mol Ther Methods Clin Dev] 2020 Nov 17; Vol. 20, pp. 169-180. <i>Date of Electronic Publication: </i>2020 Nov 17 (<i>Print Publication: </i>2021).
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