Awarded Grants
Awarded Grants
Development of a validated and patient-specific GNAO1 model for screening and testing of therapeutics
Harald Mikkers
Leiden University Medical Center, Netherlands
$100,000
Awardee: Harald Mikkers
Institution: Leiden University Medical Center, Netherlands
Grant Amount: $100,000
Funding Period: September 15, 2023 - September 14, 2024
Summary: This project will advance and improve a state-of-the-art personalized medicine tool for GNAO1. 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
Testing mechanisms and intervention strategies in GNAO1 dystonia
Kirill Martemyanov
University of Florida Scripps Institute for Biomedical Innovation and Technology
$100,000
Awardee: Kirill Martemyanov
Institution: University of Florida Scripps Institute for Biomedical Innovation and Technology
Grant Amount: $100,000
Funding Period: August 1, 2023 - July 31, 2024
Summary: This project will help advance scientific understanding about the mechanisms of dystonia. Many GNAO1 patients suffer from dystonia, commonly known as involuntary muscle movements. Bow Foundation funding will allow Dr. Martemyanov to use a mouse model to shine light on the impact of GNAO1 on dystonia and brain signals while also testing possible treatment strategies.
Personalized Anti-Sense Oligonucleotide Therapy in a Patient with GNAO1 Mutation
Jennifer Friedman
University of California San Diego and Rady Children’s Hospital
$100,000
Awardee: Jennifer Friedman
Institution: University of California San Diego and Rady Children’s Hospital
Grant Amount: $100,000
Funding Period: August 1, 2034 - July 31, 2024
Summary: Dr. Friedman partnered with the n-Lorem Foundation to support the administration of an experimental antisense oligonucleotide (ASO) medicine that targets the GNAO1 gene. Funding from the Bow Foundation will help Dr. Friedman collect and evaluate the clinical observations of this cutting-edge treatment, including changes in baseline over time and data from predetermined outcome measures. This preclinical work will allow the research team to determine if ASO treatments for other GNAO1 patients are a viable approach for other patients.