A team of doctors and scientists successfully treated rare genetic conditions with the first-ever personalized gene editing therapy. There are groundbreaking treatment results in New England Journal of Medicineaccompanying it According to a doctor who previously oversaw the FDA’s gene therapy regulation efforts.
This historical patient KJ, an infant with CPS1 deficiency, has a mortality rate of approximately 50% within the first week. Surviving patients may experience severe brain disease, mental and developmental delays, and potential liver transplants. His care team has developed personalized gene editing therapy based on CRISPR, a technology for modifying human DNA.
The success of KJ’s gene repair was successful combining previously funded medical research, including discovery of CRISPR and the discovery of CRISPR that allows mutations to be identified and human genome sequencing.
This approach to gene editing may be used in the future to assist patients with other genetic disorders, such as sickle cell disease, cystic fibrosis, Huntington’s disease, and muscular dystrophy. Although a pair of CRISPR-based drugs have already been received for sickle cell disease treatment, there is still plenty of potential to be investigated in this area.
