Gene-replacement therapy is helping children overcome fatal diseases

Replacing faulty genes with healthy ones has enabled several children with rare genetic disorders to have typical life experiences, such as attending school and living with their families

By Alice Klein

Children with some rare genetic conditions who would have once died at just a few years old could now have typical life expectancies due to gene-replacement therapy.

“We’re curing kids of fatal diseases. It’s wonderful,” says Donald Kohn at the University of California, Los Angeles.

Kohn and his colleagues are trialling a gene therapy – in which disease-causing versions of genes are replaced with normally functioning versions – for leukocyte adhesion deficiency type-1 (LAD-1).

This rare condition occurs …