UCLA researchers correct genetic mutation that causes IPEX, a life-threatening autoimmune syndrome

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.The workis described in a study published in the journal Cell Stem Cell.IPEX is caused by a mutation that prevents a gene called FoxP3 from making a protein needed for blood stem cells to produce immune cells called regulatory T cells. Regulatory T cells keep the body 's immune system in check; without them, the immune system attacks the body's own tissues and organs, which is known as autoimmu...

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