A child is a Metabolic disease He first received a test and customized treatment based on the CRisPR gene version, and he responded favorably, although it requires long -term monitoring to fully evaluate the benefits.
Intensive carbon dosage deficiency is the name of the infant born to be born as a child, which is identified K.J. In a study of publishing New England Journal of Medicine.
Baby, it He received the first level last FebruaryWhen I was between six to seven months, it was “successfully” Philadelphia Children’s HospitalThe study with the University of Pennsylvania (USA) was signed, now “It is growing well and improves”Medical Center said.
“Although KJ should be carefully monitored for the rest of his life, ours The initial results are very trusted ”, Rebecca Ahrens-Niclas and one of the signed signed by Pennsylvania Hospital.
CBS1 deficiency patients usually receive treatment with A Liver transplantation, For that they must be clinically stable, and should be old enough to intervene, but there is a risk of rapid organ failure at that time.
The team began to cooperate Read the credibility of creating customized gene version treatments In 2023, individual patients are based on the research of many years of rare metabolic disorders.
Conclusively They focused on the variation of CBS 1A rare disorder of urea cycle metabolism begins shortly after the lack of an enzyme in the liver.
Side effects
During the normal disintegration of proteins Ammonia is produced and the body makes it ureaIt is discharged by the urine, but the disease accumulates until it reaches a toxic extent that can cause damage to the brain and liver.
KJ spent the first six months of his life in the hospital The most restricted foodIt is time to take the group to design a treatment that is administered by lipid nano particles in the liver to adjust the defective enzyme.
After the initial toss, It received two more in March and April In the short term, without severe side effects, it has tolerate an increase in food proteins and requires less medications, the hospital said in a statement.
In addition, he could recover from some of the usual childhood illnesses like RhinoisWithout accumulating ammonia on his body.
There are a lot of jobs to do now, but “Researchers are cautiously confident In the evolution of the child, one of the financial providers of the study, US National Health Organizations (NIH) said.
CRISPR is an advanced gene editing technology It allows accurate changes in DNA within the cells, and the first known case of customized drug based on this technique, it is designed to go to unpacce cells so that the changes can only affect the baby.
The group expects this child to be the first of many who can benefit from one time Can be changed according to the requirements ” In each of them, Ahrens-Nicklas said in the report.
The signature of Kiran Musunuru Studio wanted other researchers to reproduce this method “For many rare diseases Provide a reasonable opportunity for many patients to live a healthy life. “
Regarding the article he did not participate, the National Biotechnology Center (CNP-CSIC) Luz Mantolie’s researcher said it was said “Precedent case” The development of temporary treatment for an individual, but “because each patient is the carrier of different mutations, is measured or globalized.”
Montolieu pointed to the science media center of science resources for journalists “Important issue” The access and affordability of this treatment for children with these diseases does not address the article.
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