Previous attempts in mice to correct a rare inherited immune disorder, called Hyper IgM X-linked immunodeficiency, have failed because standard gene therapy raised risks for cancer.Now Weill Cornell Medical College researchers believe they’ve found a way around that problem.Reporting in the July 25 issue of Nature Medicine, the investigators used a gene therapy strategy called trans-splicing to successfully correct the disease in mice without increasing malignancy risk. Trans-splicing effectively corrects the mutation at a later step in the genetic process — at the level of messenger RNA (mRNA).
