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. |
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| 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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