Thursday, December 19, 2019 Behind the Headlines: Developing Gene Therapy for Infants with a Rare, Fatal Disease Overview of the X-SCID gene therapy developed by NIAID and St. Jude scientists. 1: Blood-forming stem cells (blue) are taken from the patient and purified in the laboratory. 2: Stem cells are treated with a lentivector (green) bearing a healthy copy of the IL2RG gene (orange). 3: The patient receives a low dose of busulfan to help make room in the bone marrow for the gene-corrected stem cells. 4: The corrected stem cells are returned to the patient. Credit: NIAID Earlier this year, researchers reported that an experimental gene therapy had successfully rebuilt the immune systems of eight infants with a rare, life-threatening genetic immune deficiency disease called X-SCID. NIAID scientists and colleagues have been developing this therapy for more than a decade and also are using it to successfully treat older children and young adults with X-SCID. A new NIAID Now blog post delves into the history of this investigational treatment. National Institute of Allergy and Infectious Diseases  |  National Institutes of Health Update your subscriptions, modify your password or e-mail address, or stop subscriptions at any time on your Subscriber Preferences Page. You will need to use your e-mail address to log in. If you have questions or problems with the subscription service, please contact Subscriber Help. This service is provided to you at no charge by National Institute of Allergy and Infectious Diseases (NIAID). This email was sent to myhcistech.healthnews360@blogger.com using GovDelivery Communications Cloud on behalf of: National Institute of Allergy and Infectious Diseases · 5601 Fishers Lane · Bethesda, MD 20892 · 1-866-284-4107