Studies of aggregation-based disorders are typically associated with age-related pathology and primarily focused on extra-cellular buildup. However, intracellular aggregates or pre-oligomers may have unique disease-causing features that can be relatively overshadowed. The cytosolic presence of misfolded proteins or insoluble polyglucosan species are novel aspects of many genetic conditions as well as degenerative disorders. Therefore, this thought-provoking e-panel broadcast is designed to raise awareness, highlight new research findings, and search for commonalities among different intracellular aggregate species; including, novel treatment approaches to address aggregate-related diseases.
Topics covered include:
Comparing/contrasting a small variety of intracellular aggregates (e.g., desmin, APP, TDP-43, synucleins, polyglucosan bodies);
Identifying tissues, cell types, and/or subcellular compartments where the underlying protein or carbohydrate of interest can be found normally and/or in the disease state;
Discussing the normal role for the underlying protein or carbohydrate, and how it may aggregate in normal regenerative tissue or in diseased tissue;
Contextualizing these aggregates in terms of their disease relevance;
Determining commonalities, for instance among various intracellular aggregate clearance mechanisms;
Highlighting theoretical and ongoing translational studies for potential patient impact.
Following the panel discussion broadcast, audience members watching the live event will be able to participate in a live Q&A with the panelists. Questions can be submitted during the broadcast via Twitter or the chat room, or in advance when registering for the event.
SciTalk: Lafora Disease & Removing the Pathogenic Glycogen-like Aggregates