Custom CRISPR Therapy Paves the Way for Treating Rare Genetic Disorders in Baby
- Thy TX NGUYEN
- Jul 1
- 2 min read
In a groundbreaking achievement, doctors have helped a baby boy with a serious genetic disorder by using a specially designed gene-editing treatment. The baby, known as KJ, was born with a rare condition called CPS1 deficiency, which affects the liver's ability to remove harmful ammonia from the blood. This can lead to serious health issues, including brain damage. The new treatment, developed in just six months, is the first of its kind and provides hope for people with rare genetic diseases.
The treatment used a special tool called a "base editor," a more advanced version of the well-known CRISPR gene-editing technique. Unlike traditional CRISPR, which cuts DNA, this new method only makes small changes to the DNA, correcting a specific mistake in the gene. This approach is designed to target and fix specific genetic problems, like the one KJ has, without causing major changes to the rest of the DNA. This breakthrough brings us closer to creating personalized treatments for genetic disorders.
KJ received the gene-editing therapy through three doses delivered by lipid nanoparticles that carried the genetic instructions to his liver cells, and the results have been promising. Since starting the treatment, KJ has shown improvements: his ammonia levels are more controlled, he can eat more protein, and he needs less medicine. These results were shared at an important medical meeting and published in a top medical journal.
Though KJ's condition isn’t fully cured, the treatment has helped him avoid needing a liver transplant, which was once his only option. Doctors hope that more doses of the treatment in the future will continue to improve his health. So far, the therapy has been safe, with only mild side effects that went away on their own. These positive results offer hope for other patients who have similar genetic conditions.
This achievement marks an important step forward in personalized medicine, where treatments are specifically designed for each person's unique genetic needs. Researchers are hopeful that this gene-editing method will lead to more treatments for genetic disorders, helping many more people in the future.
Created: May 20th, 2025
Citations:
Kaiser, J. (2025, May 15). Gene-editing therapy made in just 6 months helps baby with life-threatening disease. Science. https://www.science.org/content/article/gene-editing-therapy-made-just-6-months-helps-baby-life-threatening-disease
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