Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo

Hyper-IgM1 is a rare X-linked combined immunodeficiency caused by mutations in the CD40 ligand (CD40LG) gene with a median survival of 25 years, potentially treatable with in situ CD4+ T cell gene editing with Cas9 and a one-size-fits-most corrective donor template. Here, starting from our research-...

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Detalles Bibliográficos
Autores principales: Asperti, Claudia, Canarutto, Daniele, Porcellini, Simona, Sanvito, Francesca, Cecere, Francesca, Vavassori, Valentina, Ferrari, Samuele, Rovelli, Elisabetta, Albano, Luisa, Jacob, Aurelien, Sergi Sergi, Lucia, Montaldo, Elisa, Ferrua, Francesca, González-Granado, Luis Ignacio, Lougaris, Vassilios, Badolato, Raffaele, Finocchi, Andrea, Villa, Anna, Radrizzani, Marina, Naldini, Luigi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10482894/
https://www.ncbi.nlm.nih.gov/pubmed/37693944
http://dx.doi.org/10.1016/j.omtm.2023.08.020

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