The disease has no cure.
Children affected by Batten disease are burdened by a slew of symptoms including loss of vision, impaired cognition and mobility problems. Making matters even worse, seizures and early death can occur at later stages of the disease.Professor Jonathan Cooper of Washington University School of Medicine in St Louis, one of the project’s leaders.
“That was encouraging but we needed to test the treatment in larger brains with a structure more like those of a child,” said another project leader, Professor Tom Wishart, of Edinburgh University’s Roslin Institute, where Dolly the Sheep “Sheep ovaries were collected from abattoirs, eggs were removed and fertilized. Crispr reagents were added to make the required changes in CLN1 and the eggs were then implanted into surrogate sheep,” explained Wishart.“These are symptomless carriers, like the parents of Batten disease children,” added Wishart. “From these we could then breed sheep that have two faulty copies. These go on to develop a disease like those children and became the subjects of our therapy trials.
“You can miss two crucial issues,” added Cooper. “How to deliver the drug to the right place in a bigger brain and how to scale up dosing.”
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