Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.
Journal Information
Full Title: Mol Ther Methods Clin Dev
Abbreviation: Mol Ther Methods Clin Dev
Country: Unknown
Publisher: Unknown
Language: N/A
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Subject Category: Medicine, Research & Experimental
Available in Europe PMC: Yes
Available in PMC: Yes
PDF Available: No
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"Declaration of interests A.F.M. is an inventor on patents describing liver gene transfer approaches for metabolic diseases and/or treatment of hyperbilirubinemia."
"This work was supported by an 10.13039/501100004923AFM-Téléthon grant (ID21826) to A.F.M. and by ICGEB intramural funds. We would like to thank the staff of the ICGEB adeno-associated vector unit (AVU) and the bioexperimentation facilities for technical support. We thank Viktoryia Sidarovich and Pamela Gatto of HTS and Validation Core Facility, Dep. CIBIO, University of Trento for performing ddPCR experiments."
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Last Updated: Aug 05, 2025