For the first time, scientists in the United States have attempted to edit a human gene with the hopes of curing a disease by permanently changing a person’s DNA.
As the Associated Press reports, the experiment took place yesterday on a 44-year-old patient named Brian Madeux, who suffers from a metabolic disease called Hunter syndrome. The revolutionary gene-editing process is familiar to anyone who has followed the advent of CRISPR gene-editing tech: DNA is cut open, a gene is inserted, and it’s stitched back up. The repaired DNA is then sent into the patient’s body through an IV, where it becomes a permanent part of the DNA. In Madeux’s case, he received billions of copies of a corrective gene that will hopefully prolong and dramatically improve his life.
Hunter syndrome is caused by a missing gene that makes an enzyme that breaks down certain carbohydrates. Madeux has had 26 operations over the years trying to fix some of the damage. Symptoms can be eased by weekly doses of the missing enzyme, but the costs are exorbitant ($100,000 to $400,000 a year). The hope is that by flooding Madeux’s system with cells with the missing gene, his body will start producing the enzyme on its own. His doctors should know whether the gene therapy is working in the next few months.
If it’s successful, it could be a major boost to CRISPR and the nascent field of gene therapy. As the Wall Street Journal reported in April, trials for human gene editing began in China earlier this year.ML