Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Hacein‐Bey‐Abina, Salima; Hauer, Julia; Lim, Annick; Pïcard, Capucine; P. Wang, Gary; C. Berry, Charles; Martinache, Chantal; Rieux‐Laucat, Frédéric; Latour, Sylvain; H. Belohradsky, Bernd; E. Leiva, Lily; U. Sorensen, Ricardo; Debré, Marianne; Casanova, Jean‐Laurent; Blanche, Stéphane; Durandy, Anne; D. Bushman, Frederic; Fischer, Alain; Cavazzana, Marina

doi:10.1056/nejmoa1000164

Public

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Published in New England Journal of Medicine • Jul 21, 2010

Authors:

Salima Hacein‐Bey‐Abina

Julia Hauer

Annick Lim

Abstract

BACKGROUND: The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chai...

Subject

Medicine

Severe combined immunodeficiency

Genetic enhancement

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