Kennedy Krieger Institute Researchers Receive Launch Award From State to Study Rare Pediatric Leukodystrophy

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BALTIMORE, May 26, 2023 – Kennedy Krieger Institute’s Moser Center for Leukodystrophies received more than $350,000 from the Maryland Stem Cell Research Commission (MSCRF) to fund a collaborative project that will study a form of pediatric leukodystrophy called LBSL, or leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation. This rare genetic condition progressively affects the white matter of a child’s brain and spinal cord.

The grant will allow researchers to grow three-dimensional cellular balls that resemble a human brain, called brain organoids, which will have many cell types including those that produce myelin, or the brain’s white matter.

“These cells will also be genetically modified to produce a color signal, allowing for easy analysis of their development and interaction with other cells,” said Christina Nemeth Mertz, PhD, a research scientist at the Moser Center and principal investigator of this project. “It is our goal to study these organoids to determine how and why white matter is impacted in children with LBSL.”

Other participating researchers are Ali Fatemi, MD, MBA, chief medical officer and director of the Moser Center for Leukodystrophies, and Dr. Mingyao Ying, PhD, both at the Kennedy Krieger Institute. Lena Smirnova, PhD, an assistant professor at Johns Hopkins Bloomberg School of Public Health, will also participate.

This year’s grants from the MSCRF fund the work of 39 scientists based at Maryland institutions; it is the largest amount of money the state has awarded for stem cell research since 2010.  

“This is a significant investment in our work, but also in future discoveries towards a cure for young patients with this rare disease,” Dr. Fatemi said.


About Kennedy Krieger Institute:

Kennedy Krieger Institute, an internationally known nonprofit organization located in the greater Baltimore-Washington, D.C., region, transforms the lives of more than 27,000 individuals a year through inpatient and outpatient medical, behavioral health and wellness therapies; home and community services; school-based programs; training and education for professionals; and advocacy. Kennedy Krieger provides a wide range of services for children, adolescents and adults with diseases, disorders and injuries that impact the nervous system, ranging from mild to severe. The Institute is home to a team of investigators who contribute to the understanding of how disorders develop, while at the same time pioneering new interventions and methods of early diagnosis, prevention and treatment. Visit for more information about Kennedy Krieger.     

Jessica Gregg
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