Rare disease charity funds $48,000 in CRISPR research at Michigan State University

Wednesday, June 13, 2018

East Lansing, MI – The Bow Foundation today announced a grant of $48,000 to the Michigan State University Department of Pharmacology & Toxicology. The grant will expand research into a rare genetic disorder with no common name.

The research project will be led by Dr. Richard Neubig, Chair of the Department of Pharmacology & Toxicology in the College of Osteopathic Medicine. It will use revolutionary CRISPR technology to create mouse models to study GNAO1-related neurodevelopmental gene mutations. Through the study, researchers will gather more information about GNAO1, test existing drugs, and identify possible new treatment options.

Roughly 70 children worldwide are currently known to be impacted by a GNAO1-related neurodevelopmental disorder. Children with the disease suffer from severe developmental delays, seizures and uncontrolled muscle movements.

“Dr. Neubig’s research gives the promise of hope to the dozens of GNAO1 patients around the world,” Bow Foundation co-chairs Emily Bell and Alice Fox said in a statement. “By helping the medical community better understand the impact of GNAO1-related neurodevelopmental disorders, it could also open doors to better treatment options for patients. We’re excited to fund this new research project and are thrilled to be working with Michigan State. We look forward to gaining new insights and funding additional studies as we work to increase GNAO1 awareness and change lives.” 

The Neubig lab will develop mice that carry two of the more common GNAO1 mutations. Researchers will analyze the development of these mice and undertake preclinical therapeutic trials with existing drugs to identify new options for controlling GNAO1 neurodevelopmental symptoms in human patients.

“We look forward to working with the Bow Foundation to better understand the consequences of GNAO1 mutations on child brain development and to try to identify new therapeutic options,” Dr. Neubig said. “Their support of families of children with this rare disorder and their support of research to help these children makes a huge difference.”

This is the second major Bow Foundation medical research grant. The foundation previously funded a $100,000 GNAO1 stem cell research study at the University of Virginia’s School of Medicine.

The Bow Foundation is a non-profit dedicated to supporting GNAO1 families through enhanced research and increased awareness. The foundation was launched in April of 2017 by two families who have children with a GNAO1 disorder. Visit www.BowFoundation.org to learn more or donate. 

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