Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency | Zendy

Salima HaceinBeyAbina | Zendy; Julia Hauer | Zendy; Apiradee Lim | Zendy; Capucine Pïcard | Zendy; Gary P. Wang | Zendy; Charles C. Berry | Zendy; Chantal Martinache | Zendy; Frédéric RieuxLaucat | Zendy; Sylvain Latour | Zendy; Bernd H. Belohradsky | Zendy; Lily E. Leiva | Zendy; Ricardo U. Sorensen | Zendy; Marianne Debré | Zendy; JeanLaurent Casanova | Zendy; Stéphane Blanche | Zendy; Anne Durandy | Zendy; Frederic D. Bushman | Zendy; Alain Fischer | Zendy; Marina Cavazzana | Zendy

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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Author(s) -

Salima HaceinBeyAbina,

Julia Hauer,

Apiradee Lim,

Capucine Pïcard,

Gary P. Wang,

Charles C. Berry,

Chantal Martinache,

Frédéric RieuxLaucat,

Sylvain Latour,

Bernd H. Belohradsky,

Lily E. Leiva,

Ricardo U. Sorensen,

Marianne Debré,

JeanLaurent Casanova,

Stéphane Blanche,

Anne Durandy,

Frederic D. Bushman,

Alain Fischer,

Marina Cavazzana

Publication year - 2010

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1000164

Subject(s) - medicine , severe combined immunodeficiency , genetic enhancement , immunodeficiency , hematopoietic stem cell transplantation , immunology , immune system , transplantation , leukemia , common gamma chain , gene , biology , interleukin 10 , biochemistry

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 chain.

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