For the first time in the world: Doctors in the US successfully cure an infant using individual gene editing

The team of doctors successfully applied personalised gene editing therapy for the first time. Source: Yuichiro Chino via Getty Images

A group of doctors and scientists has made a breakthrough in the treatment of a rare genetic disease by applying the first ever personalised gene editing therapy. The results of the treatment were published in the authoritative medical journal The New England Journal of Medicine.

Here's What We Know

The patient was an infant named KJ, who was born with CPS1 deficiency, a rare genetic disease with an estimated 50% mortality rate in the first week of life. Survival with this pathology is often accompanied by severe brain damage, developmental delays and even the need for a liver transplant.

Doctors have used a unique therapy based on CRISPR technology, which allows them to edit DNA directly in the patient's body. This success was made possible by many years of research and discoveries, including the decoding of the human genome and the discovery of CRISPR.

Personalised gene editing is opening up new horizons in the treatment of other hereditary diseases, such as sickle cell anaemia, cystic fibrosis, Huntington's disease and muscular dystrophy. Some CRISPR-based drugs have already received FDA approval for the treatment of sickle cell disease, but research in this area is ongoing.

This could be the beginning of a new era in medicine, where treatment will be tailored individually to each patient based on their unique genetic code.

Source: The New York Times