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

JumpstartGrant Assistance ProgramBow Foundation2025

Aug 1

Awardee: Blair Leavitt

Institution: University of British Columbia

Grant Amount: $100,000

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

Summary: Using patient-derived stem cells and a specialized mouse model, this research aims to develop a targeted correction for the R209H mutation. Lipid nanoparticle (LNP) technology will refine gene-editing delivery methods for clinical applications.

JumpStartBow Foundation2025

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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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