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Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.

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Title: Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.
Authors: Lisjak M; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Iaconcig A; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Guarnaccia C; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Vicidomini A; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Moretti L; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Collaud F; Généthon, 91000 Évry, France.; Université Paris-Saclay, Université d'Évry, Inserm, Généthon, Integrare Research Unit UMR_S951, 91000 Évry, France., Ronzitti G; Généthon, 91000 Évry, France.; Université Paris-Saclay, Université d'Évry, Inserm, Généthon, Integrare Research Unit UMR_S951, 91000 Évry, France., Zentilin L; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy., Muro AF; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Aug 28; Vol. 31, pp. 101103. Date of Electronic Publication: 2023 Aug 28 (Print Publication: 2023).
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: Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.
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  Data: <searchLink fieldCode="AU" term="%22Lisjak+M%22">Lisjak M</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Iaconcig+A%22">Iaconcig A</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Guarnaccia+C%22">Guarnaccia C</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Vicidomini+A%22">Vicidomini A</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Moretti+L%22">Moretti L</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Collaud+F%22">Collaud F</searchLink>; Généthon, 91000 Évry, France.; Université Paris-Saclay, Université d'Évry, Inserm, Généthon, Integrare Research Unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Ronzitti+G%22">Ronzitti G</searchLink>; Généthon, 91000 Évry, France.; Université Paris-Saclay, Université d'Évry, Inserm, Généthon, Integrare Research Unit UMR_S951, 91000 Évry, France.<br /><searchLink fieldCode="AU" term="%22Zentilin+L%22">Zentilin L</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Muro+AF%22">Muro AF</searchLink>; International Centre for Genetic Engineering and Biotechnology, 34149 Trieste, Italy.
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        Text: English
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              Text: 2023 Aug 28
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