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Doctors successfully treated a baby with the first ever personalized gene-editing therapy
A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy.
Results of the groundbreaking treatment have been published in The New England Journal of Medicine, with an accompanying editorial by a doctor who had previously overseen the FDA's gene-therapy regulation efforts. The successful gene repair for KJ combined years' worth of previous federally-funded medical research, including the discovery of CRISPR and human genome sequencing that allowed the mutation to be identified. A pair of CRISPR-based drugs have already received FDA approval for sickle cell disease treatments, but there is still a lot to potentially be explored in this field.
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