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

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

(AAV)-mediated episomal gene replacement therapy for monogenic liver disorders is currently limited in pediatric settings due to the loss of vector DNA, associated with hepatocyte duplication during liver growth. Genome editing is a promising strategy leading to a permanent and specific genome modif...

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Bibliographic Details
Main Authors: Giulia Bortolussi, Alessandra Iaconcig, Giulia Canarutto, Fabiola Porro, Filippo Ferrucci, Claudia Galletta, Cristian Díaz-Muñoz, Vipin Rawat, Alessia De Caneva, Olayemi Joseph Olajide, Lorena Zentilin, Silvano Piazza, Luka Bočkor, Andrés Fernando Muro
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene editing
neonatal mice
liver-directed gene editing
adeno-associated viral vector, AAV
bilirubin
CRISPR-SaCas9
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123002000

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http://www.sciencedirect.com/science/article/pii/S2329050123002000

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