For the first time, US scientists have attempted to directly edit a gene inside a living human. Completed this past Monday in California, the procedure was done on 44-year old Brian Madeux in an attempt to cure the Hunter syndrome he suffers from.
Hunter syndrome is a condition where a person lacks the gene responsible for making an enzyme that breaks down carbohydrates in the body. This causes a buildup in cells, which eventually wreaks havoc across their body. Those with Hunter syndrome suffer from frequent colds and ear infections, heart and breathing problems, skin and eye issues, bone and joint flaws, bowel issues, hearing loss, distorted facial features, and brain fog. This means the majority of people with Hunter syndrome spend their lives confined to a wheelchair, and many remain dependent on their parents until they (often) die prematurely. But, by possibly correcting the genetic structure of a patient, scientists believe they can eradicate these issues.
Using a process dubbed “zinc finger nucleases”, scientists are able to seek out and cut a specific piece of DNA. The first step is to place the corrected gene within a specially constructed virus that is not ineffective but is designed to ferry the genes safely into the body. Copies of these genes, numbering in the billions, are then inserted into the body via an IV drip. They will then make their way to the patient’s liver, where they proceed to cut the patient’s DNA and insert themselves into this newly created gap. In this case, the newly added gene will then direct the liver cells to make the enzymes the patient lacks, and the healing process can begin.
If successful, the procedure will allow Brian Madeux to lead a somewhat normal life. Though it won’t reverse any damage that’s already been done, it will stop any further issues from developing. The procedure is still highly experimental though, so only time will tell if it’s a feasible treatment. If it does prove to be a success, it means we can start treating those with Hunter disease in their infancy, well before the majority of adverse effects manifest.
Images: Wikimedia, UCSF Benioff, Sangamo