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Lead investigator

Budget: $30,000

Disorders: Charcot-Marie-Tooth Disease

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

Neuromuscular disorders are a large group of diseases that affect the proper functioning of muscles. With the revolution in gene discovery for these disorders, and the prohibitively long time needed to develop accurate mammalian models for each new variant discovered, simpler animal models are needed to bridge the gap and help guide the development of higher models. Dr. Parker’s team uses the nematode C. elegans with its powerful genetics and rapid behavioural methodologies to model aspects of human neurodegenerative diseases. Charcot-Marie Tooth (CMT) disease is a hereditary peripheral motor and sensory neuropathy, and many of the genes involved are conserved throughout evolution. Dr. Parker’s team has developed a high throughput in vivo drug screening platform using C. elegans that has led to clinical trials for human neurodegenerative diseases. Here Dr. Parker’s team are now developing C. elegans models for CMT to be used for drug discovery and development.

Impact: