Cellular gene therapies have the potential to restore missing proteins. In Fabry disease, we use modified blood cells corrected to express the missing alpha-galactosidase A enzyme, which help reduce the build up of toxic metabolites in the patient. First we harvest cells from the patient (autologous). We then modify the cells to express the missing enzyme. The resulting gene-modified cells are then reintroduced to the patient, where they can spread and distribute the missing enzyme to neighboring cells and effectively restore enzyme levels. This restoration clears toxic metabolites that would otherwise accumulate and damage critical tissues, including the kidneys, heart, and brain.

Cellular Therapy for Fabry