Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

Gene therapy clinical trials require rigorous non-clinical studies in the most relevant models to assess the benefit-to-risk ratio. To support the clinical development of gene therapy for β-thalassemia, we performed in vitro and in vivo studies for prediction of safety. First we developed newly GLOB...

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Detalles Bibliográficos
Autores principales: Lidonnici, Maria Rosa, Paleari, Ylenia, Tiboni, Francesca, Mandelli, Giacomo, Rossi, Claudia, Vezzoli, Michela, Aprile, Annamaria, Lederer, Carsten Werner, Ambrosi, Alessandro, Chanut, Franck, Sanvito, Francesca, Calabria, Andrea, Poletti, Valentina, Mavilio, Fulvio, Montini, Eugenio, Naldini, Luigi, Cristofori, Patrizia, Ferrari, Giuliana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6178212/
https://www.ncbi.nlm.nih.gov/pubmed/30320151
http://dx.doi.org/10.1016/j.omtm.2018.09.001

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