Personalized Anti-Sense Oligonucleotide Therapy in a Patient with GNAO1 Mutation

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.