Development of a validated and patient-specific GNAO1 model for screening and testing of therapeutics

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

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Unravelling HGprt functioning in fore- and midbrain development using brain organoids

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Establishing a Krabbe Disease Newborn Screening Biobank and Natural History Database Platform