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...
Na minha lista:
| Principais autores: | , , , , , , , , |
|---|---|
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer Nature
2017-07-01
|
| Colecção: | EMBO Molecular Medicine |
| Assuntos: | |
| Acesso em linha: | https://doi.org/10.15252/emmm.201707601 |
| Tags: |
Sem tags, seja o primeiro a adicionar uma tag!
|