CD34+ cells transduced with ADA retrovir

This is an autologous cell-based gene therapy developed by the National Human Genome Research Institute (NHGRI) for the treatment of severe combined immunodeficiency (SCID) caused by adenosine deaminase (ADA) deficiency. The therapy involves harvesting CD34+ hematopoietic stem cells from the patient's bone marrow or umbilical cord blood. These cells are then transduced ex vivo with a retroviral vector containing a functional copy of the human ADA gene. Once the gene is integrated, the corrected cells are infused back into the patient, where they are intended to engraft and produce functional ADA enzyme, thereby restoring immune function by allowing the development and survival of T, B, and NK lymphocytes.

Other names

Autologous CD34+ cells transduced with a human ADA geneADA gene therapy (NHGRI)