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

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
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
Series:EMBO Molecular Medicine
Subjects:
brain damage
genetic liver disease
homologous recombination
hyperbilirubinemia
kernicterus
Online Access:https://doi.org/10.15252/emmm.201707601

Internet

https://doi.org/10.15252/emmm.201707601

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