A team of doctors and scientists successfully treated rare genetic diseases with the first personalized gene editing therapy. The results of pioneering treatment already exist New England Journal of Medicineaccompanied by a doctor who previously oversees the FDA's gene therapy regulation efforts.
This historic patient is KJ, an infant with CPS1 deficiency with a mortality rate of about 50% in the first week. Patients who can survive experience severe brain disorders, psychological and developmental delays, and potential liver transplants. His nursing team has developed a personalized gene editing therapy based on CRISPR, a technology used to modify human DNA.
KJ's successful gene repair combines years of federally funded medical research, including the discovery of CRISPR and human genome sequencing, allowing the identification of mutations.
This gene editing method may be used in the future to help patients with other genetic diseases such as sickle cell disease, cystic fibrosis, Huntington's disease and muscular dystrophy. A pair of CRISPR-based drugs have been treated with sickle cell disease, but there are still many drugs to explore in this area.
