British scientists have announced plans to develop a gene therapy for motor neurone disease.
Scientist Professor Stephen Hawking is one of the best-known victims of the disease – although few victims live as long as him.
A single anonymous donation of more than £2 million from the USA has enabled researchers at Sheffield University to embark on the project.
They plan to target a gene, the SOD1 gene, which is linked to 20% of inherited cases of the disease.
According to the researchers, they hope to seek approval for trials in August next year.
Researcher Professor Mimoun Azzouz said: "This pioneering project has the potential to deliver the first meaningful neuroprotective therapy for motor neurone disease, offering a real beacon of hope for patients and their families living with this cruel disease."
Professor Pam Shaw, who founded the Sheffield Institute for Translational Neuroscience, said: "Motor neurone disease is considered a rare disease and therefore tends to be overlooked in terms of funding for the development of new therapies."
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