Pre-clinical investigation of candidate therapies for NUBPL disease

MDBRNUBPL
Written By Million Dollar Bike Ride

Awardee: Neal Mathew

Institution: Children's Hospital of Philadelphia

Grant Amount: $50,198

Funding Period: February 1, 2022 - January 31, 2023

Summary:

The overall goal of this project is to identify lead therapeutic candidates for NUBPL-/- based mitochondrial disease. We hypothesize that therapeutic modeling of NUBPL-/- genetic disease across 3 evolutionarily distinct models will enable identification and optimization of a lead therapeutic regimen to prioritize as a precision medicine that improves health in human NUBPL-/- disease patients.

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