Systems-level investigation of mucopolysaccharidosis IIIA identifies deficient synaptic activity as a key driver of disease progression
Alon M. Douek, Adrian Salavaty, Florian Kreuder, Sebastian-Alexander Stamatis, Joel R. Steele, Iresha Hanchapola, Anup D. Shah, Ralf B. Schittenhelm, Mirana Ramialison, Peter David Currie, Jan Kaslin
The paper uses a zebrafish model of MPS IIIA to identify synaptic activity deficiencies as a key driver of disease progression through systems-level omics analyses and a novel bioinformatic tool.
bioRxiv · Oct 3, 2022 · PreprintNeuroscience