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Family photo: Christian and Alysia Padilla-Vaccaro with daughters Gigi (left), Annabella (seated) and Evangelina. Evangelina was born with a rare genetic immunodeficiency disorder, but thanks to a novel gene therapy at UCLA, her immune system was restored.
Photograph of Brenden Whittaker
At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.
Led by Dr. Donald Kohn and Katelyn Masiuk, the researchers engineered a “viral vector” that would turn on the FoxP3 gene only in regulatory T cells — and not in other types of cells.
Hussein El Kerdi during his 2016 procedure at UCLA. His father, Ali El Kerdi (with cell phone) looks on.
Evangelina Vaccaro received Dr. Kohn’s treatment for bubble baby disease in 2012.
Evangelina Vaccaro (far right), who received Dr. Kohn’s treatment for bubble baby disease in 2012, with her family before her first day of school.
Headshot of Donald Kohn in a lab sitting down