Doctors at Children’s Hospital of Philadelphia have successfully used a first-of-its-kind gene-editing treatment to save a baby diagnosed with a rare and deadly metabolic blood disorder. The child, KJ, had one of the most severe forms of the condition, which affects about 1 in 1.3 million people and has a high mortality rate for infants. The treatment was tailored specifically to the baby’s genetic mutation and was developed within just six months of his birth.
Using CRISPR technology, doctors edited a specific section of his DNA and delivered modified instructions to his liver cells. He received two infusions at seven and eight months of age and by the seven-week follow-up, he was able to eat more dietary protein and cut his medication dose in half with no negative side effects. Doctors Rebecca Ahrens-Nicklas and Kiran Musunuru, who led the research, say the new technique allows for flexible updates, like a GPS system that can be reprogrammed for different genetic targets.
This personalized therapy could pave the way for rapid treatments for other rare diseases using reusable components like mRNA and nanoparticles. The study was published in “The New England Journal of Medicine” and presented at the American Society of Gene and Cell Therapy conference. The team’s next goal is to create genomic centers where patients can get individualized therapies in real time.
Source: GOOD MORNING AMERICA