The Canavan Disease Clinical Research Training Fellowship supports research toward the cause, treatment or cure of Canavan disease, a rare and fatal genetic disorder affecting the myelin sheath that insulates and protects nerves and the brain. The two-year fellowship will provide McPhee with $50,000 each year, plus a stipend for tuition reimbursement.
The Canavan Foundation's mission is to help prevent Canavan disease through education and testing and to support research efforts, which have yet to find an effective therapy. Children with Canavan disease cannot crawl or walk, may suffer from seizures or mental retardation, and cannot perform activities of daily living. Canavan disease generally results in death by 10 years of age. For more information about the Canavan Foundation, visit www.canavanfoundation.org.
McPhee plans to examine the development and genetic regulation of myelinating cells called oligodendrocytes, which form the white matter in the brain.
"I am very grateful that I have been honored with this grant, and I plan to make the very most out of this wonderful opportunity," said McPhee, who works in the Cell & Gene Therapy Center at the Medical School.
"My objectives are to determine the underlying disease mechanism in Canavan disease, and to improve our understanding of how the brain normally develops," said McPhee. "The research project will examine how certain cells in the brain develop in the setting of Canavan disease, compared to the normal fetal or newborn brain."