Post Date: September 30, 2020
Cincinnati Children’s has been awarded a five-year, $8 million federal grant to accelerate research into the rare birth condition fragile X syndrome (FXS) with the goal of translating recent lab discoveries into patient-ready treatments.
Fragile X is the most common single-gene cause of autism and the most common inherited form of developmental disability impacting 1 in 4,000 males and 1 in 6-8,000 females worldwide.
The grant, from the Eunice Kennedy Shriver National Institute of Child Health and Human Development, establishes Cincinnati Children’s and the University of Cincinnati as one of three cooperative fragile X research centers funded by the National Institutes of Health. The others are based at the Baylor College of Medicine and the Emory University School of Medicine.
The Cincinnati-based work will be led by Craig Erickson, MD, Director of the Fragile X Research and Treatment Center at Cincinnati Children’s. Since the founding of this Center in 2012, the Cincinnati Children’s Fragile X team has investigated new treatment options across age ranges with an eye to improving our understanding of how individuals with fragile X react to specific treatments under development.
The new grant will support plans to conduct parallel, synced studies in a mouse model of FXS and in humans with the syndrome.
“This work will improve our underlying understanding of fragile X biology with a strong emphasis on developing treatment strategies targeting the biological brain abnormalities discovered by the research efforts,” Erickson says.
The Research Horizons blog features news and insights about the latest discoveries and innovations developed by the scientists of Cincinnati Children's. This blog does not provide medical advice, diagnosis, or treatment.