Gene therapy restores immunity in infants with X-SCID

          The article "Gene therapy restores immunity in infants with rare immunodeficiency disease" talks about the most recent gene therapy trial that was done on infants with X-SCID. X-SCID is a rare inherited disorder that causes those infected to not develop or have normal functioning immune cells. The disease is caused by the mutation in the IL2RG gene which increases the infants susceptibility to succumb to severe infections. Scientist at NIAID and St. Jude Children's Hospital tried to restore the immune function by injecting a normal copy of the IL2RG gene into the infants stem cells. Compare to previous attempts in treating infants with X-SCID this method of gene therapy yielded better results. 7 out of the 8 infants test showed an increase in immune cells (T cells, B cells, and NK cells), while the 8th infant showed in increase in T cells. The researchers are still monitoring the infants that receive this gene treatment and are starting to enroll more infants into the trial.


     This was a very informative article. It is always a welcome news when new treatments for diseases that impact infants yields a positive result. The article talked about other methods that researchers tried to help those with X-SCID and it was very interesting to see the different outcomes.  One used chemotherapy regimens and while it did increase T cells, those cells did not have the functions an immune cell would have. It made me wonder why did the T cells generated from the gene therapy and the chemotherapy did not do the same thing, one being fully functional and the other not.


Links:
https://www.niaid.nih.gov/news-events/gene-therapy-restores-immunity-infants-rare-immunodeficiency-disease
https://www.sciencedaily.com/releases/2019/04/190417171019.htm