For the first time in the world, a child who is suffering from a rare disease receives a treatment that allows DNA to replace DNA

An American child is one of a first person Rare genetic disorder treated with crisprCustomized gene version therapy that allows scientists Edit DNA.

The child known as KJ was found after a while Disorder Sintety 1 (CBS 1) is called a serious disadvantage, which is calculated A child for a million children.

This disease is a Increased the amount of ammonia in the bloodIt can cause vomiting, inferiority, laziness, seizures, cerebral inflammation and coma. The disease is roughly killed Half of children It is affected. Treatment usually contains low protein diet until child age TranslapionBut this approach also has health risks.

KJ began to receive customized CRSPR treatment at the age of six months, which allowed his doctors Reduce the dependence on the presence of the drug According to a case inspection released on Thursday, to keep your ammonia level less.New England Journal of Medicine‘.

“Although KJ should be carefully monitored for the rest of his life, our initial results Very trusted“Dr. Rebecca Ahrens-Niclas, the director of the Filadelphia Children’s Hospital (USA) genetic treatment program.

Crispr gene version treatment person

CrisPR operates in specific genetic views, Cut DNA with accuracy Take advantage of the natural instructions for repairing the cell at those points Deactivate the harmful gene or insert the adjusted version. In this case, the treatment went to a defective gene in the KJ’s liver, cutting the DNA in the right place where the enzyme was in the right place.

According to the researchers, the success of the practice means that it can treat other patients Avant -Cart Technology. “Although KJ is a patient, we believe this is the first of many benefits that can change the needs of each patient,” Ahrens-Niclas said.

Extension challenges

KJ’s case is a trusted opinion proof, but experts warn that they are attempts to create Crispr treatmentsThey put forward many challenges. Technically, it is very difficult to use genetic editing for other organs instead of the liver. Since the total cost of the procedure is also expensive to create such treatment 700,000 exceeds eurosAccording to the Associated Press Team, even though the number approaches the price of a standard liver transplant.

Although this process helped to improve the quality of life of KJ, the research team could not fully evaluate Potential side effects Intervention for security reasons.

Dr. Alena Bans, Professor of Genetics at the University of Hertfordshire (United Kingdom) who did not participate in this practice Most diseases They As a result of various mutations in genesMore than noticeable errors with accurate versions made in CRISPR treatment.

“Crispr Applies to any disease caused by single nucleotide change; However, diseases are caused by a variety of diseases, so general strategies are more accurate than others, ”Bans said in a statement.