GENE_EDITING_colo_02
Simon Landrein
The first gene-editing treatment: 10 Breakthrough Technologies 2024
By Antonio Regalado
WHO
CRISPR Therapeutics, Editas Medicine, Precision BioSciences, Vertex Pharmaceuticals
WHEN
Now
The first gene-editing cure has arrived. Grateful patients are calling it “life changing.”
It was only 11 years ago that scientists first developed the potent DNA-snipping technology called CRISPR. Now they’ve brought CRISPR out of the lab and into real medicine with a treatment that cures the symptoms of sickle-cell disease.
Sickle-cell is caused by inheriting two bad copies of one of the genes that make hemoglobin. Symptoms include bouts of intense pain, and life expectancy with the disease is just 53 years. It affects 1 in 4,000 people in the US, nearly all of them African-American.
