Fox Chase Cancer Center News

Gene Therapy a Possible Treatment for Metabolic Condition in Children

PHILADELPHIA (December 16, 2019) – New research from Fox Chase Cancer Center has set the framework for gene therapy as a possible treatment for human cystathionine beta-synthase deficiency, the most common inborn error of metabolism found in children. “We showed that the DNA we treated was expressed in the liver and could reverse the phenotypes we observed in mice with CBS deficiency.”

VIEW STORY