A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

ABSTRACT:

Background

In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted in vector-induced leukemia through enhancer-mediated mutagenesis in 25% of patients. We assessed the efficacy and safety of a self-inactivating retrovirus for the treatment of SCID-X1.

Methods

We enrolled nine boys with SCID-X1 in parallel trials in Europe and the United States to evaluate treatment with a self-inactivating (SIN) γ-retrovirus vector containing deletions in viral enhancer sequences expressing γc (SIN-γc).

Results

All patients received bone marrow-derived CD3

SUBMITTER: Hacein-Bey-Abina S 

PROVIDER: S-EPMC4274995 | biostudies-literature | 2014 Oct

REPOSITORIES: biostudies-literature