Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4(+) T‐cells for the treatment of Hyper IgM1

Hyper IgM1 is an X‐linked combined immunodeficiency caused by CD40LG mutations, potentially treatable with CD4(+) T‐cell gene editing with Cas9 and a “one‐size‐fits‐most” corrective template. Contrary to established gene therapies, there is limited data on the genomic alterations following long‐rang...

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Detalles Bibliográficos
Autores principales: Canarutto, Daniele, Asperti, Claudia, Vavassori, Valentina, Porcellini, Simona, Rovelli, Elisabetta, Paulis, Marianna, Ferrari, Samuele, Varesi, Angelica, Fiumara, Martina, Jacob, Aurelien, Sergi Sergi, Lucia, Visigalli, Ilaria, Ferrua, Francesca, González‐Granado, Luis Ignacio, Lougaris, Vassilios, Finocchi, Andrea, Villa, Anna, Radrizzani, Marina, Naldini, Luigi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690452/
https://www.ncbi.nlm.nih.gov/pubmed/37916874
http://dx.doi.org/10.15252/embj.2023114188

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690452/
https://www.ncbi.nlm.nih.gov/pubmed/37916874
http://dx.doi.org/10.15252/embj.2023114188

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