Harald Mikkers of Leiden University Medical Center in the Netherlands will work to advance and improve a state-of-the-art personalized medicine tool for GNAO1 (Guanine Nucleotide-binding protein Alpha Other). He will use funding from the Bow Foundation to create a validated stem cell GNAO1 model that opens the doors to various drug screening efforts. The work will investigate how GNAO1 impacts neurons and evaluate the suitability of the iPSC-based model for testing of therapeutics and drug responses.
The Bow Foundation awarded a total of $300,000 in grants to fund three new projects to advance medical research into GNAO1-related disorders, a rare neurological genetic condition with no common name. The Bow Foundation grant program is administered in partnership with the Orphan Disease Center at the University of Pennsylvania.
To date, roughly 400 children around the world have been diagnosed with a GNAO1–related genetic disorder. Children with the disease suffer from a range of symptoms, including severe developmental delays, seizures and uncontrolled muscle movements.
Source: Bow foundation
