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Sickle Cell clinical trial patient Evie Junior
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
Headshot of Lili Yang at her desk
Researchers will focus on an immunotherapy known as CAR T, which uses genetically modified stem cells to target and destroy HIV-infected cells like this one.
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.
Dr. Sarah Larson (left) and Yvonne Chen
Photograph of Dr. Steven Jonas
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.