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Adenosine deaminase-transduced hematopoietic stem cells (National Human Genome Research Institute )

Hematopoietic stem cells were derived from the bone marrow of adenosine deaminase severe combined immunodeficiency disease patients (ADA SCID). These cells were retrovirally delivered with the adenosine deaminase gene.

CD34+ cells were enriched using Isolex 300i. CD34+ cells were placed in culture with flt3 ligand (300 ng/mL), stem cell factor (50 ng/mL), and MGDF (50 ng/mL). After 2 days of prestimulation, half the cells were transduced with the GCsap-M-ADA vector and half were transduced with the MND-ADA vector. Each vector included the identical ADA cDNA but slightly different vector backbones.

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Adult Stem / Progenitor Cell
Homo sapiens
Adenosine deaminase-transduced hematopoietic stem cells