From birth to gene-edited in 6 months: Custom therapy breaks speed limits

From birth to nearly a year, baby KJ’s ammonia levels have improved through gene-editing therapy, now at 35th percentile in weight while requiring ongoing monitoring for potential future liver transplants. The FDA approved the customized base-editing treatment within a week after meeting with researchers, and KJ is now managing an immune response to prevent rejection of his gene change. Though still growing slower than expected, his family and medical team remain cautiously optimistic about his progress so far.