transduced autologous lymphocytes

Transduced autologous lymphocytes (NHGRI) refer to a pioneering gene therapy approach developed by the National Human Genome Research Institute (NHGRI) for the treatment of adenosine deaminase (ADA) deficiency-related severe combined immunodeficiency (ADA-SCID). The therapy involves the ex vivo transduction of a patient's own T-lymphocytes with a functional human ADA gene using a retroviral vector. Once re-infused, these cells express the ADA enzyme, which is critical for the metabolic health and survival of infection-fighting white blood cells. This approach was the basis for the first successful human gene therapy trials in the early 1990s. While it demonstrated safety and provided partial immune restoration, it has largely been superseded by gene therapies targeting hematopoietic stem cells (CD34+ cells), which provide a more permanent and comprehensive source of ADA-producing cells. The NHGRI continues to monitor the long-term safety and efficacy of this intervention in historical cohorts through natural history studies.

Other names

ADA-transduced lymphocytesautologous lymphocytes transduced with a human ADA geneADA-transduced autologous T-lymphocytes