Transcription profiling of human CD4+ and CD8+ T-cells from gene therapy treated ADA patients and from healthy controls

ABSTRACT: Gene transfer into HSCs by gammaretroviral vectors (RV) is an effective treatment for inherited blood disorders, although potentially limited by the risk of insertional mutagenesis. We evaluated the genomic impact of RV integration in T-lymphocytes from adenosine deaminase (ADA)-Severe combined immunodeficiency (SCID) patients 10 to 30 months after infusion of autologous, genetically-corrected CD34+ cells. Expression profiling on ex vivo T-cell bulk population revealed no difference with respect to healthy controls. To assess the effect of vector integration on gene expression at the single cell level, primary T-cell clones were isolated from two patients. T-cell clones harboured either one or two vector copies per cell and displayed partial to full correction of ADA expression, purine met

ORGANISM(S): Homo sapiens

SUBMITTER: Alessandro Aiuti 

PROVIDER: E-GEOD-17354 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress