The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA.
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Incidence of transplant related mortality
Timeframe: Up to 100 days post treatment
Incidence of transplant related mortality
Timeframe: At 6 months post treatment
Transgene copy number on peripheral blood mononuclear cells (PBMCs)
Timeframe: Up to 15 years post treatment
Transgene copy number on sorted cell populations
Timeframe: Up to 15 years post treatment
Detection of replication-competent lentivirus (RCL)
Timeframe: 3 months post treatment
Absence of any severe adverse events due to insertional mutagenesis
Timeframe: Up to 15 years post treatment
Change in Artemis mRNA levels
Timeframe: At Day 0, 12 months and 24 months post treatment
Adverse events
Timeframe: Up to 15 years post treatment
Transgene copy number in the transduced CD34+ cells in the drug substance
Timeframe: At Day 0
Change in total number of T cells
Timeframe: 6, 12, 24 months post treatment
Change in distribution of different subpopulations
Timeframe: 6, 12, 24 months post treatment
Change in T lymphocyte in vitro proliferation in the presence of mitogens and antigens
Timeframe: 6, 12, 24 months post treatment
Change in repertoire of T lymphocytes
Timeframe: 12, 24 months post treatment
Evaluation of the B lymphocyte compartment
Timeframe: 6 months post treatment
Evaluation of the B lymphocyte compartment
Timeframe: At 12 months post treatment
Evaluation of the B lymphocyte compartment
Timeframe: At 24 months post treatment