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

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 in vivo...

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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: Porro, Fabiola, Bortolussi, Giulia, Barzel, Adi, De Caneva, Alessia, Iaconcig, Alessandra, Vodret, Simone, Zentilin, Lorena, Kay, Mark A, Muro, Andrés F
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2017
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Reports
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5623861/
https://ncbi.nlm.nih.gov/pubmed/28751579
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201707601
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