Article 64HAT Gene-edited sheep offer hope for treatment of lethal childhood disease

Gene-edited sheep offer hope for treatment of lethal childhood disease

by
Robin McKie, Science Editor
from on (#64HAT)

Roslin Institute engineered a flock to help research into the genetics of Batten disease

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.

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