Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Gene transfer into HSCs is an effective treatment for SCID, although potentially limited by the risk of insertional mutagenesis. We performed a genome-wide analysis of retroviral vector integrations in genetically corrected HSCs and their multilineage progeny before and up to 47 months after transpl...

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Autori principali: Aiuti, Alessandro, Cassani, Barbara, Andolfi, Grazia, Mirolo, Massimiliano, Biasco, Luca, Recchia, Alessandra, Urbinati, Fabrizia, Valacca, Cristina, Scaramuzza, Samantha, Aker, Memet, Slavin, Shimon, Cazzola, Matteo, Sartori, Daniela, Ambrosi, Alessandro, Di Serio, Clelia, Roncarolo, Maria Grazia, Mavilio, Fulvio, Bordignon, Claudio
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2007
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC1934603/
https://ncbi.nlm.nih.gov/pubmed/17671653
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI31666
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