A baby in the US is among the first people with a rare genetic disorder to be treated with CRISPR, a customised gene-editing therapy that allows scientists to edit DNA. The baby, known as KJ, was ...

The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare ...

For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation.

director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at CHOP, in a statement from the hospital. Soon after his birth last summer, KJ was diagnosed with severe carbamoyl ...