Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy

X-linked chronic granulomatous disease (CGD) is associated with defective phagocytosis, life-threatening infections, and inflammatory complications. We performed a clinical trial of lentivirus-based gene therapy in four patients (NCT02757911). Two patients show stable engraftment and clinical benefi...

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Detalles Bibliográficos
Autores principales: Sobrino, Steicy, Magnani, Alessandra, Semeraro, Michaela, Martignetti, Loredana, Cortal, Akira, Denis, Adeline, Couzin, Chloé, Picard, Capucine, Bustamante, Jacinta, Magrin, Elisa, Joseph, Laure, Roudaut, Cécile, Gabrion, Aurélie, Soheili, Tayebeh, Cordier, Corinne, Lortholary, Olivier, Lefrere, François, Rieux-Laucat, Frédéric, Casanova, Jean-Laurent, Bodard, Sylvain, Boddaert, Nathalie, Thrasher, Adrian J., Touzot, Fabien, Taque, Sophie, Suarez, Felipe, Marcais, Ambroise, Guilloux, Agathe, Lagresle-Peyrou, Chantal, Galy, Anne, Rausell, Antonio, Blanche, Stephane, Cavazzana, Marina, Six, Emmanuelle
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9975109/
https://www.ncbi.nlm.nih.gov/pubmed/36706754
http://dx.doi.org/10.1016/j.xcrm.2023.100919

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