A small group of scientists, regulators, business people and patient advocates met recently at the National Academy of Sciences in Washington, D.C., to discuss the path forward in using genome editing tools, like CRISPR, to modify the human germline – that is, eggs, sperm or fertilized eggs. Such a change is heritable, passed to future generations.
It was the inaugural meeting of the International Commission on the Clinical Use of Human Germline Genome Editing. The tone during the day [August 13] was considerably more measured than the media splash that Victoria Gray, a 34-year-old from Mississippi with severe sickle cell disease, had made a few weeks earlier.
She's the first person to undergo a treatment using CRISPR, but the clinical trial she's in, sponsored by Vertex Pharmaceuticals, targets only cells that give rise to red blood cells, which shed their DNA as they mature. Victoria is the first of 45 patients expected to enroll, with results in summer 2022.
Altering the germline is a different story.
To continue reading, go to Genetic Literacy Project, where this post first appeared.