National Institute on Aging 5R01AG070209-05

Using Genetic Diversity to Manage Neurological Disease

Award for research on genetic diversity to manage neurological diseases, focusing on rare genetic neurodegenerative diseases and therapeutic interventions, funded by the National Institute on Aging.

Description

The project focuses on understanding the mechanism behind and treatment of genome abnormalities causing rare genetic neurodegenerative diseases. The research involves Gaussian process regression machine learning to study genetic variation in the Niemann-Pick C1 (NPC1) gene. The project aims to develop a deep genome-based understanding of proteostasis for therapeutic intervention. The project involves developing small molecules to regulate the Hsp70 axis to correct NPC1 variants. The award is funded by the National Institute on Aging, with a focus on aging and neurodegenerative diseases. The project duration is from January 1, 2021, to December 31, 2026.

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