Towards a Treatment for Leukodystrophy Using Cell-Based Interception and Precision Medicine

Abstract

Cell-based interception and precision medicine is a novel approach aimed at improving healthcare through the early detection and treatment of diseased cells. Here, we describe our recent progress towards developing cell-based interception and precision medicine to detect, understand, and advance the development of novel therapeutic approaches through a single-cell omics and drug screening platform, as part of a multi-laboratory collaborative effort, for a group of neurodegenerative disorders named leukodystrophies. Our strategy aims at the identification of diseased cells as early as possible to intercept progression of the disease prior to severe clinical impairment and irreversible tissue damage.

Keywords

• cell-based interception and precision medicine
• leukodystrophy
• POLR3-related leukodystrophy (POLR3-HLD)
• proteomics
• single-cell technologies
• SCoPE2-MS