Correcting FA mutations in all cells of the body may prevent issues such as bone marrow failure and cancer in people with FA. Since mutations in FA genes start during the gestational process, the ideal time to correct genes may be in utero. The goal of this study is to use laboratory-based experiments to determine whether gene editing in utero (during gestation) can correct FA gene variants in various tissues of the body.
Researchers: Agnieszka Czechowicz