autologous CD34+ cells transduced with MND-ADA retroviral vector

This autologous cell therapy consists of CD34+ hematopoietic stem and progenitor cells (HSPCs) derived from the patient, which are genetically modified ex vivo using a gamma-retroviral vector. The vector, specifically the MND-ADA construct, utilizes the MND promoter (a synthetic promoter derived from the myeloproliferative sarcoma virus) to drive the expression of the human adenosine deaminase (ADA) gene. Developed primarily by researchers at UCLA and Children's Hospital Los Angeles, this therapy targets Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID). By integrating a functional ADA gene into the patient's stem cell genome, the therapy aims to restore endogenous ADA enzyme production, facilitating the maturation of functional T, B, and NK cells and providing long-term immune reconstitution. While historically significant, retroviral approaches in this space have largely transitioned toward lentiviral platforms to improve safety and transduction efficiency.

Other names

ADA-SCID retroviral gene therapyKohn ADA gene therapyautologous CD34+ cells transduced with MND-ADA