Advantages of Gene Therapy to Children with Immunodeficiency
Three separate clinical trials published in The New England Journal of Medicine in 2021 found that 48 children with severe combined immunodeficiency (SCID) who accepted a corrected gene delivered by a virus have functioning immune systems after two to three years of receiving the treatment.
What is SCID?
SCID, often referred to as bubble baby or bubble boy disease, is brought on by genetic changes in the ADA gene, which produces the immune system’s adenosine deaminase enzyme. These mutations stop the immune system from developing normally, and patients typically pass away within one to two years of birth. The condition has been treated with bone marrow transplants, although the process is risky and not always possible with qualified donors. Antibodies and antibiotics are frequently administered twice weekly as a stop-gap measure, while gene treatments promise to address the underlying cause of the issue.
How Gene therapy is done
Among 50 infants and toddlers with SCID caused by ADA mutations, a gene therapy was explored in three separate Phase 1/2 trials, one in the UK and two in the US (ADA-SCID). Before injecting the cells back into the kids, the researchers took blood cells from the patients and tweaked a lentivirus to transport a healthy version of ADA. 48 of the patients have immune systems that can successfully combat invasive infections, such as COVID-19, two to three years later.
