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Scientists Develop Gene Therapy for Rare Immunodeficiency Disorder

Scientists report that they have achieved success in identifying a gene therapy for the rare genetic disorder, SCID-X1, which is a severe combined immunodeficiency disease, NPR’s Shots blog reports. Without the therapy, the disease is “mostly uniformly fatal” but until now, therapies have been linked to the development of several types of blood cancer. “Researchers give each child between 2 million and 10 million genetically engineered stem cells after the gene that they lack, called the IL-2 common gamma chain receptor gene, has been inserted into a mouse virus” and “once the gene-corrected cells are infused back, they multiply and stimulate the production of normal immune cells.” The latest gene therapy experiment used a “self-inactivating” virus to deliver the corrective gene to avoid the development of leukemia.

Read the article published by NPR’s Shots blog.