Last week a 44-year-old California man was the first to be implanted with billions of copies of "a corrective gene and a genetic tool to cut his DNA in a precise spot," according to The Guardian. The patient, Brian Madeux, suffers from Hunter syndrome and has endured 26 operations to treat symptoms of the disease. Dr. Chester Whitley of Masonic Cancer Center, a Consortium member, is a lead scientist researching the therapy. It is based on zinc-finger nucleases, which work like "molecular scissors that seek and cut a specific piece of DNA." Dr. Whitley is quoted in the Atlantic, where he notes, “You know exactly where you’re going in the genome. It’s not like using a shotgun hoping you’re hitting a bird. It’s like using a rifle.” While still in clinical trial, if proven effective, zinc-finger nucleases could "kick off a new era for gentic disorders, one where kids never have to suffer their effects in the first place."
Wednesday, November 22, 2017