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Gene-replacement therapy is helping children overcome fatal diseases

Changing defective genes with wholesome ones has enabled a number of kids with uncommon genetic issues to have typical life experiences, reminiscent of attending faculty and residing with their households


27 Could 2022

A inventory picture of an individual holding a toddler’s hand in hospital

Shutterstock / Pammy Studio

Youngsters with some uncommon genetic situations who would have as soon as died at only a few years outdated may now have typical life expectations on account of gene-replacement remedy.

“We’re curing youngsters of deadly illnesses. It’s fantastic,” says Donald Kohn on the College of California, Los Angeles.

Kohn and his colleagues are trialling a gene remedy – by which disease-causing variations of genes are changed with usually functioning variations – for leukocyte adhesion deficiency type-1 (LAD-1).

This uncommon situation happens …

Article amended on 30 Could 2022

This text has been amended to appropriate the date the infants with X-SCID began receiving gene remedy.

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