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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| Hoofdauteurs: | , , , , , , , , |
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| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Springer Nature
2017-07-01
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| Reeks: | EMBO Molecular Medicine |
| Onderwerpen: | |
| Online toegang: | https://doi.org/10.15252/emmm.201707601 |
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