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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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Bibliographic Details
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

Description
ISSN:	2329-0501
DOI:	10.1016/j.omtm.2023.08.022