(COLUMBUS, Ohio) – The Rosenau Family Research Foundation has awarded a three-year grant of $375,000 total in funding to Allison Bradbury, MS, PhD, along with collaborators at Cincinnati Children’s Hospital Medical Center and Auburn University, for studying in-utero gene therapy as a potential treatment for Krabbe disease and other lysosomal storage diseases.
“While it is already known that gene therapy is a viable approach for Krabbe disease, hurdles in treatment still exist,” said Dr. Bradbury, who is a principal investigator in the Jerry R. Mendell Center for Gene Therapy at Nationwide Children’s. “There is a significant need to treat Krabbe disease before symptom onset, and our research aims to get closer to meeting that need.”
Because the onset of Krabbe disease occurs in utero, comprehensive treatment can be challenging. With that in mind, Dr. Bradbury’s lab will evaluate in-utero gene therapy in a preclinical model of Krabbe disease using an identical gene therapy construct that has previously been evaluated in a postnatal setting.
Krabbe disease, also known as globoid cell leukodystrophy, is a rare neurodegenerative disease that progresses rapidly, causing premature death by 2 years of age. Symptoms include loss of motor skills, hearing, vision, and voluntary movement, and seizures. Krabbe disease was recently voted on by the Federal Newborn Screening Advisory Committee to be included on the Recommended Uniform Screening Panel.
About The Abigail Wexner Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2024-25 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children’s to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.