autologous CD34+ cells transduced with retroviral vector encoding adenosine deaminase (GOSH)

This investigational ex vivo gene therapy was developed by Great Ormond Street Hospital (GOSH) for the treatment of adenosine deaminase (ADA) deficiency-related severe combined immunodeficiency (ADA-SCID). The therapy involves the collection of a patient's own (autologous) CD34+ hematopoietic stem cells, which are then genetically modified using a gammaretroviral vector to incorporate a functional copy of the human human adenosine deaminase gene. These modified cells are reinfused into the patient, where they engraft in the bone marrow and give rise to immune cells that produce the ADA enzyme, thereby restoring immune function. While GOSH was a pioneer in these gammaretroviral trials, the institution later transitioned to using more advanced lentiviral vectors in collaboration with other research centers to improve safety and efficacy.

Other names

GOSH ADA gene therapyGammaretroviral ADA gene therapyautologous CD34+ cells transduced with retroviral vector encoding adenosine deaminase-Great Ormond Street Hospital for Children NHS Foundation Trust-adenosine deaminase deficiency