Hopes raised for nerve disease cure within 10 yrs
Published: March 14, 2005
A treatment for a common form of motor neuron disease could be available within ten years using a new technique that can switch off the faulty genes responsible in mice, scientists said yesterday.
Genetic forms of amyotrophic lateral sclerosis (ALS), the wasting condition that afflicts Professor Stephen Hawking, could be treatable using an advanced gene-silencing technique called RNA interference (RNAi), new research on mice has suggested.
At present there is no cure for any form of motor neuron disease, in which the nerve cells that control the muscles degenerate and die. About 5,000 patients in Britain are affected by it, most of whom die within two to five years of diagnosis.
Between 5 and 10 per cent of cases are inherited and 20 per cent of these are linked to mutations in a single gene known as SOD1.
It is this gene that has been successfully targeted in the new research. Research teams in Britain and Switzerland have used RNAi to turn it off in mice genetically engineered with the ALS mutation, and then used gene therapy to replace a healthy copy of SOD1 instead.
While the work is in its infancy and much more research is needed ahead of human trials, it suggests that similar techniques might be useful as a therapy for the disease in people.
“This is the first proof of principle in the human form of a disease of the nervous system in which you can silence the gene and at the same time produce another normal form of the protein,” said Patrick Aebischer of the Federal Polytechnic School in Lausanne, a leader of the Swiss study.
Dr Aebischer said it would probably take a decade to produce therapies, but that the technique might also be useful in other diseases of the nervous system. “I would not be surprised to see, in the next ten years, this technology used for treating diseases of the nervous system, such as inherited forms of Parkinson’s disease or Huntington’s disease,” he said.
The British work was led by Scott Ralph of the bio- technology company Oxford Biomedica. Both sets of results are published today in the journal Nature Medicine.
RNAi is one of the most recent and promising advances in medical science, providing a powerful tool with which to turn off faulty genes that cause disease.
It works by exploiting the cellular maintenance process by which genes make proteins with important functions. When a protein is made, the DNA in a cell’s nucleus creates a set of instructions written in messenger RNA, a genetic signalling chemical. This then transfers the genetic code for a protein to the part of the cell in which it is needed.
In RNAi, short pieces of double-stranded RNA are introduced to the cell, where they bind with messenger RNA carrying particular instructions. This causes the cell to see the messenger RNA as faulty and destroy it, so the genetic blueprint is not delivered and the protein is not made.
“This is the first demonstration of therapeutic efficacy of RNA interference-mediated gene silencing in an ALS model,” said Cedric Raoul, a lead author of the Swiss study.
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