Baby Saved by Gene-Editing Therapy ‘Graduates’ from Hospital
A 10-month-old baby boy named KJ has made a remarkable recovery after receiving the first-of-its-kind gene-editing treatment for his rare genetic disease. Diagnosed with carbamoyl-phosphate synthetase 1 deficiency, a condition that affects only one in 1.3 million people and kills 50% of babies by early infancy, KJ was treated with CRISPR technology at the Children’s … Read more