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| Scientists deploy GM sheep in fight to treat Huntington’s disease | Scientists deploy GM sheep in fight to treat Huntington’s disease |
| (about 14 hours later) | |
| Robin McKie Observer science editor | Robin McKie Observer science editor |
| Sat 8 Jul 2017 22.00 BST | Sat 8 Jul 2017 22.00 BST |
| Last modified on Wed 14 Feb 2018 21.34 GMT | |
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| Scientists at Cambridge University have co-opted an unusual ally in their battle to find treatments for an incurable degenerative ailment that affects thousands of people in the UK. They have taken charge of a flock of merino sheep that have been genetically modified to carry the gene for Huntington’s disease. | Scientists at Cambridge University have co-opted an unusual ally in their battle to find treatments for an incurable degenerative ailment that affects thousands of people in the UK. They have taken charge of a flock of merino sheep that have been genetically modified to carry the gene for Huntington’s disease. |
| The research, led by neuroscientist Professor Jenny Morton, aims to understand how to pinpoint early symptoms of the brain condition, which affects more than 6,700 people in the UK. | The research, led by neuroscientist Professor Jenny Morton, aims to understand how to pinpoint early symptoms of the brain condition, which affects more than 6,700 people in the UK. |
| The gene responsible for Huntington’s was isolated more then 30 years ago but scientists have yet to develop drugs that might halt or even slow its development in patients. The brain’s complexity has defied attempts to understand how the condition develops. | The gene responsible for Huntington’s was isolated more then 30 years ago but scientists have yet to develop drugs that might halt or even slow its development in patients. The brain’s complexity has defied attempts to understand how the condition develops. |
| “Until now, much of our effort has been based on research on mice or rats,” said Morton. “But sheep should make better research subjects. Not only do they live much longer than rodents, their brains are larger and closer in size and structure to humans.” | “Until now, much of our effort has been based on research on mice or rats,” said Morton. “But sheep should make better research subjects. Not only do they live much longer than rodents, their brains are larger and closer in size and structure to humans.” |
| Huntington’s disease, which affects men and women equally, is an inherited neurological condition whose symptoms manifest themselves in adulthood, usually between 35 and 55. Initially mood, personality, coordination and memory are affected but, as the disease progresses, speech, swallowing and motor function deteriorate until death occurs 10 to 25 years after symptoms first appear. There is no known cure for Huntington’s disease although there are treatments to manage symptoms. | Huntington’s disease, which affects men and women equally, is an inherited neurological condition whose symptoms manifest themselves in adulthood, usually between 35 and 55. Initially mood, personality, coordination and memory are affected but, as the disease progresses, speech, swallowing and motor function deteriorate until death occurs 10 to 25 years after symptoms first appear. There is no known cure for Huntington’s disease although there are treatments to manage symptoms. |
| Individuals with a parent affected by Huntington’s have a 50-50 chance of inheriting the gene. Charles Sabine, a former TV war correspondent and spokesman for Huntington’s patients, highlights how the disease affects families. “My father died of the condition and so did my half-brother. My brother is now in the final stages of the disease. I also carry the gene. So I am the next on the conveyor belt,” he told the Observer. | Individuals with a parent affected by Huntington’s have a 50-50 chance of inheriting the gene. Charles Sabine, a former TV war correspondent and spokesman for Huntington’s patients, highlights how the disease affects families. “My father died of the condition and so did my half-brother. My brother is now in the final stages of the disease. I also carry the gene. So I am the next on the conveyor belt,” he told the Observer. |
| In the past the condition has often been misdiagnosed or ignored in affected families, Sabine said. Men and women passed on the condition because they were unaware they were carriers. However, in 1983 scientists pinpointed the Huntington gene and tests can now identify carriers. This led to hopes that therapies to cure Huntington’s or delay its onset would be developed. Despite three decades of effort, however, these have been unsuccessful. | In the past the condition has often been misdiagnosed or ignored in affected families, Sabine said. Men and women passed on the condition because they were unaware they were carriers. However, in 1983 scientists pinpointed the Huntington gene and tests can now identify carriers. This led to hopes that therapies to cure Huntington’s or delay its onset would be developed. Despite three decades of effort, however, these have been unsuccessful. |
| “Even though we have known for decades now exactly which genetic mutation causes Huntington’s disease, we’re arguably still no nearer a cure – the best we can do is manage the symptoms,” said Morton. “Even those treatments are limited to some of the motor symptoms.” | “Even though we have known for decades now exactly which genetic mutation causes Huntington’s disease, we’re arguably still no nearer a cure – the best we can do is manage the symptoms,” said Morton. “Even those treatments are limited to some of the motor symptoms.” |
| Most research has focused on mice and rats which have been genetically modified to carry the Huntington gene. However, the short lifespan of rodents – around two years – makes it hard to model the condition’s slow impact on the human brain. By contrast, the creation of transgenic sheep that carry the gene should help the development of treatments. Sheep typically live for 10 to 12 years and their brain structures are far closer to those of humans. | Most research has focused on mice and rats which have been genetically modified to carry the Huntington gene. However, the short lifespan of rodents – around two years – makes it hard to model the condition’s slow impact on the human brain. By contrast, the creation of transgenic sheep that carry the gene should help the development of treatments. Sheep typically live for 10 to 12 years and their brain structures are far closer to those of humans. |
| The sheep to be studied by Morton were created by neuroscientist Richard Faull and geneticist Russell Snell at Auckland University and imported to Britain this year. Half the flock carry the Huntington gene, the other half do not. | The sheep to be studied by Morton were created by neuroscientist Richard Faull and geneticist Russell Snell at Auckland University and imported to Britain this year. Half the flock carry the Huntington gene, the other half do not. |
| “We will run trials and tests to monitor how the disease affects our sheep,” said Morton. It is hoped to gain a more detailed picture from these tests of how cognitive decline progresses and so pave the way for drugs to slow its impact. | “We will run trials and tests to monitor how the disease affects our sheep,” said Morton. It is hoped to gain a more detailed picture from these tests of how cognitive decline progresses and so pave the way for drugs to slow its impact. |
| “It’s an encouraging development,” said Sabine. “This gives us genuine hope that at last animal models will have relevance to Huntington’s patients like myself.” | “It’s an encouraging development,” said Sabine. “This gives us genuine hope that at last animal models will have relevance to Huntington’s patients like myself.” |
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