Development of base editing for gene therapy of elane-mutated severe congenital neutropenia

The University of Massachusetts Medical School was awarded a $753,700 Project Grant from the Department of the Army Medical Command under the Military Medical Research and Development federal grant program (CFDA 12.420). The grant will fund the development of base editing for gene therapy of ELANE-mutated severe congenital neutropenia from June 1, 2022 to May 31, 2025.

The Military Medical Research and Development program supports basic, applied, and advanced biomedical research with the goals of improving military and public health care through innovative medical solutions. This award will advance those objectives by supporting the University of Massachusetts Medical School's research into developing new gene therapy techniques using base editing to treat severe congenital neutropenia caused by mutations in the ELANE gene. The research has the potential to transform treatment for this condition, benefiting both military service members and the general public.