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.
鈥淲e鈥檙e curing kids of fatal diseases. It鈥檚 wonderful,鈥 says at the University of California, Los Angeles.
Kohn and his colleagues are trialling a gene therapy 鈥 in which disease-causing versions of genes are replaced with normally functioning versions 鈥 for leukocyte adhesion deficiency type-1 (LAD-1).
This rare condition occurs when immune cells called leukocytes…
Article amended on 30 May 2022
This article has been amended to correct the date the infants with X-SCID started receiving gene therapy.
