A flock of gene-edited sheep has been used by scientists to identify a promising treatment for a fatal brain disease. According to the researchers, their work could lead to the development of drugs to treat the disease.
Between 100 and 150 children and young adults are affected by the disease in the UK, which is caused by two symptomless parents.
Children who have two copies of the faulty gene are at risk of having vision, cognitive and mobility issues. There areSeizures and Early Death. One of the project's leaders said the effect on families is devastating.
Working with colleagues at Collaborations Pharmaceuticals, researchers showed that mice with one form of Batten disease, known as CLN1 disease, could be treated with a missingidase.
"That was encouraging but we needed to test the treatment in larger brains with a structure more like a child," said another project leader, Professor Tom Wishart. It's not possible to generalize from mouse experiments to humans. It's important to have an intermediate large model.
The project scientists used Crispr-Cas9 to modify the faulty gene in sheep. Eggs were removed and fertilised from sheep's ovaries. Eggs were implanted into surrogate sheep and Crispr reagents were used to make the required changes. A small flock of sheep were created by the scientists, each carrying a functional copy of the CLN1 gene.
The parents of children with a disease are symptomless. We could breed faulty sheep from these. The subjects of our therapy trials were the children who went on to develop a disease.
Children with healthy CLN1 genes die of this form of Batten disease. The lysosomal performance of their bodies is impaired without it. In Batten disease, this process is not possible.
The research showed that injecting the missing enzyme into the brain made a difference. It was not practical to jump straight to trials on humans.
Cooper said you can miss two important issues. The drug can be delivered to the right place in a bigger brain.
Experiments on sheep with faulty CLN1 genes gave answers. There are many telltale signs of the disease that affects humans. By calculating an appropriate dose and the route to deliver it to brains of sheep, the team was able to observe improvements in their disease.
The scientists say that the results are promising but that more research is needed to improve treatment.
The insights we have gained will help in the development of therapies for children. His point was supported by another person. We have a long way to go, but we have taken a big step.
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