Differential Tractography: A Biomarker for Neuronal Function in Neurodegenerative Disease.

ABSTRACT: GM1 gangliosidosis is an ultra-rare inherited neurodegenerative lysosomal storage disorder caused by biallelic mutations in the GLB1 gene. GM1 is uniformly fatal and has no approved therapies, although clinical trials investigating gene therapy as a potential treatment for this condition are underway. Novel outcome measures or biomarkers demonstrating the longitudinal effects of GM1 and potential recovery due to therapeutic intervention are urgently needed to establish efficacy of potential therapeutics. One promising tool is differential tractography, a novel imaging modality utilizing serial diffusion weighted imaging (DWI) to quantify longitudinal changes in white matter microstructure. In this study, we present the novel use of differential tractography in quantifying the progre

SUBMITTER: Lewis CJ 

PROVIDER: S-EPMC11451749 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature