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Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.

Archive ouverte | Benjelloun, Fatine | CCSD

International audience. Patients with mutations in the Artemis gene display a complete absence of T- and B lymphocytes, together with increased cellular radiosensitivity; this leads to a radiosensitive severe combin...

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Archive ouverte | Hacein-Bey-Abina, Salima | CCSD

International audience. Previously, several individuals with X-linked SCID (SCID-X1) were treated by gene therapy to restore the missing IL-2 receptor gamma (IL2RG) gene to CD34+ BM precursor cells using gammaretrov...

Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients.

Archive ouverte | Lagresle-Peyrou, Chantal | CCSD

International audience. Severe combined immunodeficiency (SCID) caused by mutation of the recombination-activating gene 1 (RAG1) or Artemis gene lead to the absence of B- and T-cell differentiation. The only curativ...

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