Doctors and scientists have achieved a historical first. The team successfully treated a baby’s rare disease using personalized gene-editing therapy.
Genetic diseases that can affect infants are among the greatest fears for parents. Now, a study conducted by a group of doctors and scientists has revealed promising results in this area. A US-based team has successfully treated a rare genetic disorder using a previously unheard-of method.
In this study, researchers used personalized gene-editing therapy to treat the disease, marking a world first. The study findings were published in The New England Journal of Medicine.
Baby Cured of High-Risk Disease Thanks to Gene-Editing Therapy
The baby in this historic case, named “KJ,” had a very rare metabolic disorder called CPS 1 deficiency (carbamoyl phosphate synthetase 1 deficiency). Children with this disease faced mortality rates of up to 50% in the first week. For those who survived, there were risks of severe brain problems, mental and developmental delays, and the need for liver transplantation.
The research team developed a CRISPR-based, personalized gene-editing therapy specifically for this baby, a technology that directly edits human DNA. This allowed a mutation in the patient’s DNA to be corrected using CRISPR technology, leading to successful outcomes.
This achievement has the potential to be groundbreaking as a world first. In the future, we may see more personalized gene-editing therapies. This technology could be used to treat many different types of diseases and save lives.
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