Promoterless gene targeting without nucleases rescues lethality of a Crigler‐Najjar syndrome mouse model

Abstract Crigler‐Najjar syndrome type I (CNSI) is a rare monogenic disease characterized by severe neonatal unconjugated hyperbilirubinemia with a lifelong risk of neurological damage and death. Liver transplantation is the only curative option, which has several limitations and risks. We applied an...

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Bibliographic Details

Published in:	EMBO Molecular Medicine
Main Authors:	Fabiola Porro, Giulia Bortolussi, Adi Barzel, Alessia De Caneva, Alessandra Iaconcig, Simone Vodret, Lorena Zentilin, Mark A Kay, Andrés F Muro
Format:	Article
Language:	English
Published:	Springer Nature 2017-07-01
Subjects:	brain damage genetic liver disease homologous recombination hyperbilirubinemia kernicterus
Online Access:	https://doi.org/10.15252/emmm.201707601