First Personalized Gene Therapy Using Base Editing Shows Promising Results in Baby with Rare Disorder

Gene editing helps baby with rare disease thrive

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

Baby in the US becomes the first treated with personalized CRISPR Gene-Editing

In a groundbreaking advancement in genetic medicine, a baby in the United States has become the first person to be treated with a personalized CRISPR gene-editing therapy, marking a historic moment for science and the future of rare disease treatment. According to a report by ScienceAlert, the infant was born with a rare and deadly […]

It is a genetic disease that affects one in a million children in the world. Today, it smiles at life. The Philadelphia Children's Hospital has just succeeded in a world first. The doctors have repaired the DNA of the little KJ, thanks to a technique invented by Emmanuelle Charpentier. The French woman had received the Nobel Prize in Chemistry in 2020. It is a prowess that gives hope to millions of people. (Health and well-being).

For the first time, a child with a life-threatening hereditary disease has been treated by surgically precise gene therapy. However, the little "KJ" still has to hope for healing.

For the first time, a futuristic gene therapy has helped a sick baby. The new technique allowed doctors to modify the DNA of a baby...

International researchers have called the treatment a promising medical milestone.