adenosine deaminase + peripheral blood stem cells

Adenosine deaminase + peripheral blood stem cells refers to a combination therapy used primarily for the treatment of adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID). This approach involves the use of hematopoietic stem cells (CD34+ cells) collected from mobilized peripheral blood, which are either transplanted directly (as in allogeneic hematopoietic cell transplantation) or genetically modified ex vivo to express functional adenosine deaminase before autologous transplantation. The mechanism relies on restoring ADA activity and reconstituting immune function by providing healthy or gene-corrected hematopoietic progenitors capable of differentiating into functional immune cells. This strategy is considered when enzyme replacement therapy is insufficient or unavailable, and it can be performed as an allogeneic transplant from a donor or as an autologous gene therapy product using lentiviral vectors[1][2][3][4][5].