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...

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Detalles Bibliográficos
Autores principales: Yang, Yang, Wang, Lili, Bell, Peter, McMenamin, Deirdre, He, Zhenning, White, John, Yu, Hongwei, Xu, Chenyu, Morizono, Hiroki, Musunuru, Kiran, Batshaw, Mark L., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2016
Materias:
Article
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

Internet

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

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