CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.

Authors:
Bortolussi G; Iaconcig A; Canarutto G; Porro F; Ferrucci F and 9 more

Journal:
Mol Ther Methods Clin Dev

Publication Year: 2023

DOI:
10.1016/j.omtm.2023.101161

PMCID:
PMC10716028

PMID:
38094199

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Journal Information

Full Title: Mol Ther Methods Clin Dev

Abbreviation: Mol Ther Methods Clin Dev

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Medicine, Research & Experimental

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

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Evidence found in paper:

"Declaration of interests G.B. and A.F.M. are inventors of patents describing liver gene transfer approaches for metabolic diseases and/or treatment of hyperbilirubinemia. The remaining authors declare no competing interests."

Evidence found in paper:

"The work was supported 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 Marco Baralle for proofreading this manuscript."

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Last Updated: Aug 05, 2025