A new study conducted by US researchers suggests that gene therapy can be used to treat people with severe combined immunodeficiency (SCID-X1), Youth Health reports.
The study's findings will be presented in the annual meeting of the American Society of Hematology from December 5-8 in Florida.
Although SCID-XI is a common immune-deficiency disorder, the genetic condition occurs only in 1 per 100,000 newborns.
The National Institute of Allergy and Infectious Diseases (NIAID) combined low-dose chemotherapy and gene therapy to treat five people with the immune-deficiency disorder, whose ages ranged from 7 to 24 years. All the five patients had already received stem cell transplant from their parents without much improvement in their condition.
For the study, the researchers extracted stem cells from the parents and used the Lentivirus as a vector. They manipulated the virus gene to deliver the right gene to the cell. The patients were then administered low-dose chemotherapy to get rid of the defective blood cells. Once the stem cells had the correct IL2RG gene, they were then infused into the body.
All the patients who received this treatment showed noticeable immune changes after the transplant. The first two who received the gene therapy treatment showed great improvement even in their clinical status, although one of them died due to a pre-existing condition.
