A flock of gene-edited sheep has been used by scientists to pinpoint a promising treatment for a lethal inherited brain disease that afflicts young children. The researchers, based in the UK and US, say their work could lead to the development of drugs to alleviate infantile Batten disease.
In the UK, Batten disease affects between 100 and 150 children and young adults and is inherited from two symptomless parents who each carry a rare recessive gene mutation.
