Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy

Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vectors (LVs) has emerged as a promising therapeutic option for...

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Detalles Bibliográficos
Autores principales: Jofra Hernández, Raisa, Calabria, Andrea, Sanvito, Francesca, De Mattia, Fabiola, Farinelli, Giada, Scala, Serena, Visigalli, Ilaria, Carriglio, Nicola, De Simone, Maura, Vezzoli, Michela, Cecere, Francesca, Migliavacca, Maddalena, Basso-Ricci, Luca, Omrani, Maryam, Benedicenti, Fabrizio, Norata, Rossana, Rancoita, Paola Maria Vittoria, Di Serio, Clelia, Albertini, Paola, Cristofori, Patrizia, Naldini, Luigi, Gentner, Bernhard, Montini, Eugenio, Aiuti, Alessandro, Mortellaro, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7791081/
https://www.ncbi.nlm.nih.gov/pubmed/33010230
http://dx.doi.org/10.1016/j.ymthe.2020.09.030

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