Zebrafish could be key to treating spinal muscular atrophy, say scientists

18:14 • 17.12.13

Scientists in Singapore are a step closer to finding a way to treat a spinal disease that currently has no known cure, the http://www.channelnewsasia.com reports.

Spinal muscular atrophy attacks nerve cells, and affects one in every 6,000 births globally -- and the clue to the treatment could lie in a small freshwater fish.

They may be small, but their contribution to science is far from tiny. Researchers have spent the last five years studying the neuron behavior of the zebrafish, a species of freshwater fish.

They inject a substance into the embryo of the fish so that their genes behave like those of the spinal disease.

Dr Kelvin See, a research fellow at the Genome Research institute of Singapore at A*STAR, said: "We place the live embryos under a powerful microscope that uses lasers to excite the fish so that we can see fluorescents in the motor neurons, which will then give us a level of activity found in the neurons."

Scientists have long known that the spinal disease is caused by deficiencies in the Survival Motor Neuron (SMN) gene and through their observations, they singled out low activity levels in a gene called neurexin 2.

Scientists believe that restoring the activity levels of neurexin 2 could partially recover neuron function in these infected embryos.

One of the reasons scientists use zebra fish instead of other animals like mice, is that the fish has a much simpler nervous system. Also, the embryo of the fish is transparent -- this allows scientists to study the fish while it is still alive.

Researchers said the long-term goal is to use neurexin 2 to develop a drug to treat the disease.

Associate Professor Christoph Winkler, from the department of biological sciences at the National University of Singapore, said: "We need to check whether our genes are regulated in a similar way in human patients. And if you have confirmed this, it will probably take at least five years or even longer, to develop a therapeutic strategy or drug."

The scientists aim to conduct further tests to ensure neurexin 2 is indeed the gene to zoom in on.
 

Zebrafish could be key to treating spinal muscular atrophy, say scientists