May 23, 2022. UCLA researchers have developed a base editing approach for patients born with the rare genetic immune disorder CD3 delta severe combined immunodeficiency (CD3 delta SCID). Babies with CD3 delta SCID can’t properly form T cells that fight against bacteria and disease, leaving them vulnerable if left untreated.
Grace McAuley from the UCLA lab of Dr. Donald Kohn shared preclinical results during a presentation at the American Society of Gene & Cell Therapy’s 2022 Annual Meeting on May 17. Their study found that base editing can correct the mutation that causes CD3 delta SCID and enable the production of functional T cells. If this new therapy is successful in a phase 1 clinical trial, it may be used for a variety of other Primary Immunodeficiencies, opening new doors for treatment. This research was funded by the Jeffrey Modell Foundation.
In addition to this exciting scientific development, Dr. Kohn is also currently caring for one of our “Roots & Wings” babies, Arturo Garcia, at UCLA. Learn more about the research in Dr. Kohn’s lab and read the full story here: stemcell.ucla.edu/news/ucla-researchers-develop-base-editing-approach-rare-genetic-immune-disorder.