Efficacy of gene therapy for X-linked severe combined immunodeficiency.

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 chain.

Methods

The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-mediated transfer of gamma chain to autologous CD34+ bone marrow cells between 1999 and 2002. We assessed clinical events and immune function on long-term follow-up.

Results

Eight patients were alive after a median follow-up period of 9 years (range, 8 to 11). Gene therapy was initially successful at correcting immune dysfunction in eight of the nine patients. However, acute leukemi

SUBMITTER: Hacein-Bey-Abina S 

PROVIDER: S-EPMC2957288 | biostudies-literature | 2010 Jul

REPOSITORIES: biostudies-literature