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A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

artículo científico publicado en 2014

Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center

scientific article published on 23 April 2019

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

artículo científico publicado en 2018

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

artículo científico publicado en 2007

Gene therapy: X-SCID transgene leukaemogenicity

artículo científico publicado en 2006

Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management

artículo científico publicado en 2009

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

artículo científico publicado en 2011

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

artículo científico publicado en 2008

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

scientific article published on 24 April 2007

Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency

artículo científico publicado en 2014

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