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. To get the normal copy of the FoxP3 gene to the proper place within the blood stem cells, the researchers used a tool called a viral vector—a specially modified virus that can carry genetic information to a cell's nucleus without causing a viral infection. The UCLA team engineered the viral vector used in the study so that the gene is turned on only in regulatory T cells, but not in other types of cells. Read more .

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