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
A stock image of a person holding a child’s hand in hospital
Shutterstock / Pammy Studio
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 …