Published on: 2025-05-17 06:34:09
Image by Getty / Futurism Treatments Scientists used CRISPR tech to create a custom-made drug targeted at helping a newborn baby fight a rare genetic disorder. According to an extraordinary new paper published in The New England Journal of Medicine, the drug worked — marking the first time that a patient was successfully healed with a bespoke gene-hacked medicine, and a possible watershed moment for the field of genetic medicine. The baby, named KJ Muldoon, was born at the Children's Hospital
Keywords: drug genetic kj liver treatment
Find related items on AmazonPublished on: 2025-05-17 14:00:23
In the boy's fourth month, researchers were meeting with the Food and Drug Administration to discuss regulatory approval for a clinical trial—a trial where KJ would be the only participant. They were also working with the institutional review board (IRB) at Children’s Hospital of Philadelphia to go over the clinical protocol, safety, and ethical aspects of the treatment. The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month
Keywords: kj month researchers therapy treatment
Find related items on AmazonPublished on: 2025-05-17 19:00:23
In the boy's fourth month, researchers were meeting with the Food and Drug Administration to discuss regulatory approval for a clinical trial—a trial where KJ would be the only participant. They were also working with the institutional review board (IRB) at Children’s Hospital of Philadelphia to go over the clinical protocol, safety, and ethical aspects of the treatment. The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month
Keywords: kj month researchers therapy treatment
Find related items on AmazonPublished on: 2025-05-18 07:10:56
A pivotal medical milestone has been reached. For the first time ever, researchers have used a personalized CRISPR-based gene therapy to treat an infant’s rare and life-threatening illness. Doctors at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine detailed their achievement in a study published Thursday in the New England Journal of Medicine. The treated child, named KJ, was born with a metabolic disorder known to kill up to 50% of children in their infancy. Now, three months
Keywords: cps1 gene kj medicine therapy
Find related items on AmazonPublished on: 2025-05-19 19:23:07
A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy. Results of the groundbreaking treatment have been published in The New England Journal of Medicine, with an accompanying editorial by a doctor who had previously overseen the FDA's gene-therapy regulation efforts. The patient in this historic case was KJ, an infant born with CPS1 deficiency, which has about a 50 percent mortality rate within the first week.
Keywords: crispr editing gene human kj
Find related items on AmazonPublished on: 2025-05-19 17:06:06
Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation. The investigators who led the effort to save KJ are presenting their work on Thursday at the annual meeting of the American Society of Cell & Gene Therapy, and are also publishing it in the New England Journal of Medicine. The implications of
Keywords: gene kj new therapy treatment
Find related items on AmazonPublished on: 2025-05-19 22:06:06
Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation. The investigators who led the effort to save KJ are presenting their work on Thursday at the annual meeting of the American Society of Cell & Gene Therapy, and are also publishing it in the New England Journal of Medicine. The implications of
Keywords: gene kj new therapy treatment
Find related items on AmazonPublished on: 2025-05-20 07:00:00
Last August, KJ Muldoon was born with a potentially fatal genetic disorder. Just six months later, he received a Crispr treatment designed just for him. Muldoon has a rare disorder known as CPS1 deficiency, which causes a dangerous amount of ammonia to build up in the blood. About half of babies born with it will die early in life. Current treatment options—a highly restrictive diet and liver transplantation—aren’t ideal. But a team at the Children’s Hospital of Philadelphia and Penn Medicine w
Keywords: ammonia crispr gene kj treatment
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