Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

BACKGROUND & AIMS: Genome editing technology has immense therapeutic potential and is likely to rapidly supplant contemporary gene addition approaches. Key advantages include the capacity to directly repair mutant loci with resultant recovery of physiological gene expression and maintenance of d...

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Detalles Bibliográficos
Autores principales: Ginn, Samantha L., Amaya, Anais K., Liao, Sophia H.Y., Zhu, Erhua, Cunningham, Sharon C., Lee, Michael, Hallwirth, Claus V., Logan, Grant J., Tay, Szun S., Cesare, Anthony J., Pickett, Hilda A., Grompe, Markus, Dilworth, Kimberley, Lisowski, Leszek, Alexander, Ian E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7005564/
https://www.ncbi.nlm.nih.gov/pubmed/32039406
http://dx.doi.org/10.1016/j.jhepr.2019.100065

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