Doctors have successfully treated a baby with the very first Personalized Geno Edition therapy

A team of doctors and scientists successfully treated a rare genetic disease with the very first personalized genetic publishing therapy. The results of revolutionary treatment were The New England Journal of MedicineWith an accompanying doctor who had previously supervised the regulatory efforts of the FDA genes.

The patient of this history was KJ, an infant born with a deficiency in CPS1, which has a mortality rate of around 50% in the first week. Patients who survive may suffer from severe brain disease, mental delays and development and potential liver transplants. His care team has developed a personalized genetic edition treatment based on CRISPR, a technology to modify human DNA.

The repair of successful genes for KJ combined the years of previous medical research funded by the federal government, including the discovery of CRISPR and the sequencing of the human genome which made it possible to identify the transfer.

This approach to gene editing could potentially be used in the future to help patients with other genetic disorders, such as sickle cell anemia, cystic fibrosis, Huntington disease and muscle dystrophy. A pair of CRISPR drugs has already received for sickle cell treatments, but there is still a lot to explore in this area.