Holy shit
Under the radar.
KJ Muldoon was born with a rare genetic disorder, CPS1 deficiency, that is often fatal for newborns and can leave those who survive with mental and developmental delays. However, KJ recently became the first patient to have a custom gene-editing treatment to address the disorder, according to his doctors at Children’s Hospital of Philadelphia. After three rounds of infusions, the treatment appears to have been successful, and doctors are preparing to release him from the hospital. The novel approach was custom-designed for KJ’s illness, using CRISPR gene-editing technology to pinpoint the mutation in his DNA that caused the disorder. While the technology is still being studied and developed for other use cases in humans, medical experts say KJ’s case could represent a breakthrough to fix mutations in other places on a person’s DNA. The New York Times has the story.
- Tangle News
