NEW ORLEANS — Greetings from ASGCT, where the gene therapy field had some great news for once, about a highly photogenic infant.
Baby KJ gets a custom CRISPR edit to his genome
Kiran Musunuru, a researcher at the University of Pennsylvania, announced on stage Thursday that he and collaborators had, for the first time, reached into the genome of a severely ill child and rewritten the unique misspelling in his DNA.
The custom CRISPR treatment, built for just a single infant named KJ, was a landmark in the 50-year quest to read and repair the code of life. Musunuru and his collaborators, along with outside researchers and advocates, hope they’ve provided a blueprint for treating other children and adults facing deadly diseases caused by unique or ultra-rare mutations.
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