Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to cure a disease
The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot
Weekly IV doses of the missing enzyme can ease some symptoms, but cost $100,000 to $400,000 a year and don’t prevent brain damage.
Gene editing won’t fix damage he’s already suffered, but he hopes it will stop the need for weekly enzyme treatments.
The therapy has three parts: The new gene and two zinc finger proteins. DNA instructions for each part are placed in a virus that’s been altered to not cause infection but to ferry them into cells. Billions of copies of these are given through a vein.
They travel to the liver, where cells use the instructions to make the zinc fingers and prepare the corrective gene. The fingers cut the DNA, allowing the new gene to slip in. The new gene then directs the cell to make the enzyme the patient lacked.
Only 1 percent of liver cells would have to be corrected to successfully treat the disease, said Madeux’s physician and study leader, Dr. Paul Harmatz at the Oakland hospital.
Zinc finger nucleases is a different gene editing tool than CRISPR.
I originally wanted to wait the 3 months necessary to verify if this procedure worked, but this is history in the making, with enormous implications, and I want to have H+ to have it on the record.
I’ll update this article with the results of the therapy once they are disclosed.