
Mutant Sheep Are Being Bred in Lab To Fight Lethal Child Brain Disease (theguardian.com) 53
In the UK, Batten disease affects between 100 and 150 children and young adults. It is inherited from two symptomless parents who each carry a rare recessive gene mutation. That mutation interferes with the operation of lysosomes, which act as the waste disposal systems for cells. Children who carry two copies of the faulty gene begin to suffer loss of vision, impaired cognition and mobility problems. Seizures and early death follow. "It is devastating for families," Wishart said. There are several types of Batten disease, he added. "One of the more rapid types is CLN1, and that's what we have recreated in our sheep." The Roslin scientists used the gene-editing technique Crispr-Cas9 to create the faulty CLN1 gene in the sheep. "We collected sheep embryos from the abattoir," Wishart said. "Then we fertilised them and added Crispr reagents to alter their genetic structure before implanting the embryos into a surrogate sheep's uterus."