We've been playing around with our bodies through experimental gene therapy since the '80s as a radical way to either fight or prevent disease. They haven't always been effective, but the techniques to insert genes into your body have gotten better decade by decade. But in 2017 we made have pulled off a new sci-fi level procedure that will help us edit our DNA at a much faster level than before.
Brian Madeux is a 44-year-old man who deals with Hunter's syndrome, a life-threatening disease. It's caused when a gene is missing and doesn't produce an enzyme that breaks down certain carbohydrates. When your body doesn't break those down, things get really bad.
A protein structure called a "zinc finger" works as a scissor of sorts on your DNA. It opens up your DNA, places new genes in, then zips it up again. It does all this with no visible change to the DNA.
Madeux's DNA was rewritten at Oakland's UCSF Benioff Children's Hospital. It was designed to work as soon as it hit his liver cells. They tried it on mice first, but this is the first time it's been tried on a human.
Madeux was connected to an IV drip for three hours by researchers from Sangamo Therapeutics. They are a nearby biopharmaceutical company that specializes in gene editing. Their goal is in trying to make a gene therapy technique based on precision.
This new kind of gene-editing makes it so that there's no way to fix any mistake that might have been made. There have been protections set in place to keep patients like Madeux safe. The animal tests beforehand were positive and got the go-ahead from the National Institutes of Health to approve the studies.
This booming field of gene therapy can potentially correct genetic diseases at their source. Recent years have seen massive advancements in therapy with the use of genetic engineering tools like CRISPR. That has been effective in curing muscular dystrophy in animal trials and mutations in human embryos.
11. This Isn't The First Time They've Used This Technique
The same editing tools used to cure Madeux of Hunter's syndrome has been used before. Sangamo used it to edit the immune cells of HIV patients. The difference was that it was like more traditional gene therapy procedures where the cells are made outside the body.
12. What's The End Goal To Using Real-Time Gene Editing?
Madeux's trial run is just the beginning of Sangamo's attempts at these types of therapies. They are running two other clinical trials in the US to see if this real-time gene-editing trick works. They're working on two different conditions - hemophilia and Hurler-Scheie syndrome.
The gene editing won't fix the damage that Hunter's syndrome has already done to his body. It will hopefully stop his need for weekly enzyme treatments for the missing one from the gene he doesn't have. The current cost of his enzyme treatment is astronomical - $100,000 to $400,000 a year.