Cargando…
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Many genetic liver diseases present in newborns with repeated, often lethal, metabolic crises. Gene therapy using non-integrating viruses such as AAV is not optimal in this setting because the non-integrating genome is lost as developing hepatocytes proliferate(1,2). We reasoned that newborn liver m...
| Autores principales: | , , , , , , , , , , , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2016
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786489/ https://www.ncbi.nlm.nih.gov/pubmed/26829317 http://dx.doi.org/10.1038/nbt.3469 |