Harnessing the power of zebrafish to fight disease

Zebrafish

Our pioneering zebrafish research has paved the way for new treatments for a common genetic disorder, Charcot Marie Tooth disease (CMT).

CMT is the most common genetic disorder affecting the nervous system and there is currently no cure. More than 20,000 people in the UK suffer from the disease, which typically causes progressive weakness and long-term pain in the feet, leading to walking difficulties.

Research conducted by Dr Andrew Grierson and his team has revealed that zebrafish could hold the key to finding new therapeutic approaches to treat the condition.

Dr Grierson, from the University's Department of Neuroscience, said: "We have studied zebrafish with a genetic defect that causes CMT in humans. The fish develop normally, but once they reach adulthood they start to develop difficulties swimming.

"By looking at the muscles of these fish we have discovered that the problem lies with the connections between motor neurons and muscle, which are known to be essential for walking in humans and also swimming in fish."

He added: "Motor neurons are the largest cells in our bodies, and as such they are highly dependent on a cellular transport system to deliver molecules through the long nerve cell processes which connect the spinal cord to our muscles.

"We already know that defects in the cellular transport system occur early in the development of diseases such as Alzheimer's disease, Motor Neuron Disease and spastic paraplegia. Using our zebrafish model we have found that similar defects in transport are also a key part of the disease process in CMT."

Dr Grierson and his team are now seeking funding to identify new treatments for CMT using the zebrafish model.

By looking at the muscles of these fish we have discovered that the problem lies with the connections between motor neurons and muscle, which are known to be essential for walking in humans and also swimming in fish

Dr Andrew Grierson, Department of Neuroscience

The research was conducted at the University's Sheffield Institute for Translational Neuroscience (SITraN) and the MRC Centre for Developmental and Biomedical Genetics (CDBG).

SITraN is at the forefront of research, expertise and pioneering new treatments for neurodegenerative diseases. The £18-million centre houses state-of-the-art laboratories and equipment, including a clinical database of over 1,500 patients and the largest resource of human brain-bank material in the world.

CDBG brings together geneticists with clinician scientists, creating a focus of expertise in the development of animal models of human disease. The aim of its work is to stimulate the translation of findings from model systems to the development of novel therapies and clinical practice.