Gene-replacement therapy means some children who once would have died at an early age or required a gruelling stem cell transplant are now having typical life experiences, such as attending school and living at home with their families
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.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 .Subscribe now for unlimited access
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