Fixing a Fatal Genetic Defect in Babies With a Bit of Genetic Modification
1 min read
Summary
An infant in the US suffering from CPS1 deficiency, an inherited genetic condition which affects the liver, has received what is thought to be the first successful genetic therapy using CRISPR gene editing.
The condition, which affects around one in 360,000 infants and has a fatality rate of 50% within the first year, saw the child undergo intensive treatment and require a liver transplant.
CRISPR 2.0 was used to treat the patient, editing the genes in the liver cells and fixing the faulty genes.
The infant, who had been showing signs of liver failure, is now showing progressive health, although it will not be known for certain to be a success until the child ultimately avoids a liver transplant.
This success signals a step forward in personalised genetic treatments.
