Jacqui Wise , 2025-05-19 14:31:00
What has happened?
US doctors have developed and safely delivered a customised gene editing therapy to treat a baby boy with carbamoyl phosphate synthetase 1 (CPS1) deficiency, diagnosed soon after birth. The condition, which affects only one in 1.3 million people, is characterised by an inability to fully break down byproducts from protein metabolism in the liver. This causes a build up of ammonia to toxic levels that can cause severe damage to the liver and brain. Treatment includes a low protein diet until the child is old enough to receive a liver transplant. However, during the waiting period there is a risk of rapid organ failure. The disorder is estimated to kill about half of those children affected in early infancy.
What is CRISPR?
CRISPR (clustered regularly interspaced short palindromic repeats) is a technology that allows precise changes to DNA …
