Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome

Precise correction of the CD40LG gene in T cells and hematopoietic stem/progenitor cells (HSPC) holds promise for treating X‐linked hyper‐IgM Syndrome (HIGM1), but its actual therapeutic potential remains elusive. Here, we developed a one‐size‐fits‐all editing strategy for effective T‐cell correctio...

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Detalles Bibliográficos
Autores principales: Vavassori, Valentina, Mercuri, Elisabetta, Marcovecchio, Genni E, Castiello, Maria C, Schiroli, Giulia, Albano, Luisa, Margulies, Carrie, Buquicchio, Frank, Fontana, Elena, Beretta, Stefano, Merelli, Ivan, Cappelleri, Andrea, Rancoita, Paola MV, Lougaris, Vassilios, Plebani, Alessandro, Kanariou, Maria, Lankester, Arjan, Ferrua, Francesca, Scanziani, Eugenio, Cotta‐Ramusino, Cecilia, Villa, Anna, Naldini, Luigi, Genovese, Pietro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7933961/
https://www.ncbi.nlm.nih.gov/pubmed/33475257
http://dx.doi.org/10.15252/emmm.202013545

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