Huntington’s disease pigs created in hope of gene editing breakthrough

Read the original article by Henry Bodkin at here.

Scientists have engineered the the world’s first pigs with Huntington’s disease in a development that could lead to a breakthrough for humans with the condition.

The joint Chinese-American team used a genetic editing technique to introduce a segment of the human gene which causes the neurodegenerative disease.

Generating Huntington’s in a large mammal is thought to be crucial for finding a cure for the condition, which affects around 5,700 people in Briton.

Up to now, scientists have worked mainly on mice, but these do not display the full range of symptoms suffered by humans, and treatments which appeared promising on in the rodents have been found to be non-transferable.

By contrast, the pigs have been showing respiratory difficulties, which resemble those experienced by humans with HD and are not seen in mouse with Huntington’s.

Crucially, the research teams at Jinan University and Emory University will be able to test wether it is possible to use the CRISPR-Cas9 gene editing technqiue – which has been likened to taking a pair of scissors to the genetic code – to edit out Huntington’s before trying it on humans.

“We think the pig model will fill an important gap,” said co-senior author Li Shihua, professor of human genetics at Emory University.

“In pigs, the pattern of neurodegeneration is almost the same as in humans, and there have been several treatments tested in mouse models that didn’t translate to humans.”

Symptoms of Huntington’s usually become evident between 30 and 50 and can include difficulty concentrating, clumsiness, involuntary jerking and problems swallowing.

The disease is progressive and full-time nursing care is needed in it’s latter stages.

Sufferers rarely survive more than 20 years after symptoms begin and children of patients have a 50 per cent chance of inheriting the responsible gene.

The new research is published in the journal Cell.

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