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Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action

Recombinant adeno-associated virus (AAV) is an effective platform for therapeutic gene transfer; however, tissue-tropism differences between species are a challenge for successful translation of preclinical results to humans. We evaluated the use of in vitro primary hepatocyte cultures to predict in...

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Detalles Bibliográficos
Autores principales: Liu, Su, Razon, Lisa, Ritchie, Olivia, Sihn, Choong-Ryoul, Handyside, Britta, Berguig, Geoffrey, Woloszynek, Jill, Zhang, Lening, Batty, Paul, Lillicrap, David, Agrawal, Vishal, Cortesio, Christa, Gebretsadik, Kahsay, Akeefe, Hassibullah, Colosi, Peter, Kim, Benjamin, Bunting, Stuart, Fong, Sylvia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9204658/
https://www.ncbi.nlm.nih.gov/pubmed/35782594
http://dx.doi.org/10.1016/j.omtm.2022.05.008