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

Hacein‐Bey‐Abina, Salima; Garrigue, Alexandrine; P. Wang, Gary; Soulier, Jean; Lim, Annick; Morillon, Estelle; Clappier, Emmanuelle; Caccavelli, Laure; Delabesse, Éric; Beldjord, Kheïra; Asnafi, Vahid; Macintyre, Elizabeth; Dal Cortivo, Liliane; Radford, Isabelle; Brousse, Nicole; Sigaux, François; Moshous, Despina; Hauer, Julia; Borkhardt, Arndt; H. Belohradsky, Bernd; Wintergerst, Uwe; Velez, María; E. Leiva, Lily; U. Sorensen, Ricardo; M. Wulffraat, N.; Blanche, Stéphane; D. Bushman, Frederic; Fischer, Alain; Cavazzana, Marina

doi:10.1172/jci35700

Public

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

Published in Journal of Clinical Investigation • Aug 6, 2008

Authors:

Salima Hacein‐Bey‐Abina

Alexandrine Garrigue

Gary P. Wang

Abstract

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 gammaretroviral vectors. While 9 of 10 patients were successfully treated, 4 of the 9 developed T cell leukemia 31-68 mo...

Subject

Genetic enhancement

Cancer research

Insertional mutagenesis

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