When CRISPR was first introduced as a gene-editing tool in 2012, the world was in awe of all the possibilities it held — eventually earning its discoverers the Nobel Prize in Chemistry in 2020. Now, after years of refining the technology and running clinical trials, gene-editing has taken a major leap toward personalized medicine.
As Kiran Musunuru, professor of Translational Research in Penn’s Perelman School of Medicine, put it in a press statement, “The promise of gene therapy that we’ve heard about for decades is coming to fruition, and it’s going to utterly transform the way we approach medicine.”
Musunuru and a team from the Children’s Hospital of Philadelphia and Penn Medicine have successfully treated a baby boy with a rare metabolic disorder using personalized CRISPR therapy. Their case study, published in The New England Journal of Medicine, demonstrates the powerful potential of precision gene editing for rare diseases.
Ironically, ...
