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

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

Abstract 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...

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Bibliographic Details
Main Authors: Valentina Vavassori, Elisabetta Mercuri, Genni E Marcovecchio, Maria C Castiello, Giulia Schiroli, Luisa Albano, Carrie Margulies, Frank Buquicchio, Elena Fontana, Stefano Beretta, Ivan Merelli, Andrea Cappelleri, Paola MV Rancoita, Vassilios Lougaris, Alessandro Plebani, Maria Kanariou, Arjan Lankester, Francesca Ferrua, Eugenio Scanziani, Cecilia Cotta‐Ramusino, Anna Villa, Luigi Naldini, Pietro Genovese
Format: Article
Language:English
Published: Wiley 2021-03-01
Series:EMBO Molecular Medicine
Subjects:
CRISPR‐Cas gene editing
hematopoietic stem cells
T‐cell therapy
truncated EGFR
X‐linked hyper‐IgM Syndrome
Online Access:https://doi.org/10.15252/emmm.202013545

Internet

https://doi.org/10.15252/emmm.202013545

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