UCLA Researchers Correct Genetic Mutation that Causes IPEX, a Life-Threatening Autoimmune Syndrome
Los Angeles, CA, USA – UCLA researchers led by Dr. Donald Kohn have created a method for modifying blood stem cells to reverse the genetic mutation that causes a life-threatening autoimmune syndrome called IPEX. The gene therapy, which was tested in mice, is similar to the technique Kohn has used to cure patients with another immune disease, severe combined immune deficiency, or SCID, also known as bubble baby disease.