Breakthrough in Gene Editing to Cure Rare Diseases

Scientists from the USA have for the first time cured a child with a rare genetic disorder by editing their genes after birth. The baby was born with a CPS1 deficiency, which could lead to developmental delays in the brain and a 50% chance of death, but doctors were able to identify the necessary mutation and "rewrite the DNA" with a custom-made drug introduced into the liver. In the future, this technology could also be used to treat other rare diseases, including muscular dystrophy.