Gomez-Ospina, an assistant professor of pediatrics and genetics, has received a grant of $375,000 from the Rosenau Family Research Foundation to develop a stem cell treatment for Krabbe disease.

Krabbe disease is a severe genetic disorder that disrupts myelin production, resulting in the progressive loss of brain cells. The disease is most commonly diagnosed in the patient’s first year of life; without intervention, survival beyond age 2 is extremely rare. The only available treatment is allogeneic hematopoietic stem cell transplantation, which provides limited benefits, highlighting the need for more effective therapies.

Gomez-Ospina’s team will use the grant to develop a groundbreaking therapy for Krabbe disease that focuses on editing the patient’s own stem cells. Their goal is to identify the safest and most effective gene editing protocols in human stem cells and test their effectiveness in mouse models of Krabbe disease, with the goal of advancing to clinical trials.

The Rosenau Family Research Foundation’s mission is to improve the lives of people impacted by Krabbe disease and cystic fibrosis through research funding and disease advocacy.