A Gene-Editing Breakthrough Saves Infant With Rare Disease
An infant in Philadelphia has been successfully treated with a customized CRISPR gene-editing therapy for a rare and deadly disease. The infant lacked an enzyme in his liver that normally converts ammonia to urea. This caused ammonia to build up and damage organs in the body. Before the treatment, the infant was in the eighth or ninth percentile for weight - after, he is now in the 35th to 40th percentile. The infant still needs medication, but now needs less. His treatment could potentially be used as a template for creating individualized treatments quickly for other patients with rare diseases.