Development of second generation AAV gene therapy for GNAO1 deficiency

JumpstartGrant Assistance ProgramBow Foundation2025
Written By Million Dollar Bike Ride

Awardee: Miguel Sena-Esteves

Institution: University of Massachusetts

Grant Amount: $100,000

Funding Period: August 1, 2025 - July 31, 2026

Summary: This study utilizes gene silencing and replacement strategies with the innovative BI-hTFR1 capsid to enable efficient brain-wide gene delivery, paving the way for future clinical trials in GNAO1 therapy.

Million Dollar Bike Ride
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Pre-clinical development of a CRISPR-mediated gene therapy for GNAO1 Encephalopathy using patient stem cells and a mouse model
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