The Rory Belle Foundation

• Open to:Academics

• Budget: Project based

The Rory Belle Foundation is providing seed funding to support several critical components of basic, clinical and translational research on neurological disorders associated with genetic mutations in the NARS1 gene with a particular emphasis on the topics outlined below: - Basic understanding of canonical and noncanonical functions of NARS1 - Development and characterization of NARS1 Disorder models including, but not limited to: patient-derived cell models of NARS1 Disorder (ex. iPSCs, neurons, reporter lines), animal models, other models (yeast, drosophila) - Identification of biomarkers for NARS1 Disorder using models and patient samples - Validation of preliminary therapeutic compounds able to restore or improve NARS1 Disorder - Development of novel therapeutic strategies for NARS1 Disorder - Design and implementation of a natural history study for NARS1 Disorder to validate or develop relevant clinical outcome measures - Novel proposals for NARS1 Disorder