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LEMS-induced disruption of the nanoscale organization of the active zone

Many neuromuscular diseases result in functional disruptions at the neuromuscular junction (NMJ); the site of communication between motor neurons and muscle fibers. Lambert-Eaton Myasthenic Syndrome (LEMS) is caused when a patient’s own immune system attacks and removes calcium channels from transmitter release sites at the NMJ, causing muscle weakness. Previously, the simple loss of calcium channels was thought to result in the muscle weakness seen in LEMS, but recently it has become apparent that the pathophysiology of LEMS is more complicated. In fact, the organization of many proteins at the neurotransmitter release site is significantly disrupted in LEMS, indicating a more complex pathophysiology. Unfortunately, exploring the specific proteins whose organization is disrupted in LEMS has not been possible because of the technical limitations of imaging these very small regions of the NMJ. However, recent advances in super-resolution microscopy can now provide the resolution necessary to observe the changes that occur at transmitter release sites in LEMS. This proposal aims to identify the specific proteins that are altered in LEMS, which will provide new potential targets for the development of alternative therapies. The data generated from this project will also enhance our understanding of neurotransmitter release sites at the NMJ, which may prove beneficial for the development of treatments for other neuromuscular diseases.
https://doi.org/10.55762/pc.gr.156997
Grantee: Tyler Tarr, Ph.D.
Grant type: Development Grant
Award total: $208,212
Institution: University of Pittsburgh
Country: Pennsylvania, United States