Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Completed
National Heart, Lung, and Blood Institute (NHLBI)
Phase 1/Phase 2
2013-08-02
The aim of this study is to assess the safety and efficacy of autologous transplantation of
hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID
infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The
level of gene transfer in blood cells and immune function will be measured as endpoints.
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Completed
National Human Genome Research Institute (NHGRI)
Phase 1/Phase 2
2013-08-02
The aim of this study is to assess the safety and efficacy of autologous transplantation of
hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID
infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The
level of gene transfer in blood cells and immune function will be measured as endpoints.
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
Completed
National Institute of Allergy and Infectious Diseases (NIAID)
Phase 1/Phase 2
2013-08-02
The aim of this study is to assess the safety and efficacy of autologous transplantation of
hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID
infants and children following human ADA cDNA transfer by the EFS-ADA lentiviral vector. The
level of gene transfer in blood cells and immune function will be measured as endpoints.
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