Axonal organelle transport in Niemann-Pick Type C
My work focuses on axonal organelle transport in healthy and diseased neurons. Our goal is to uncover mechanisms that contribute to altered axonal homeostasis in NPC using live-neuron imaging combined with genetic and pharmacologic approaches.
Key Points:
- Proper transport is important in maintaining neuron growth, survival, and function but is particularly challenging with axons that can extend up to one meter in length
- Lysosomes must navigate the cytoplasm appropriately to efficiently coordinate events throughout the cell
- Axonal accumulation of organelles is common to both mouse and human NPC neuropathology, and precedes symptom-onset and neurodegeneration in NPC mice
This work was supported by the Intramural Research Program of the National Institute of Neurological Disorders and Stroke, NIH.