from the Nu-Skin dept.
A child has been given a new genetically modified skin that covers 80% of his body, in a series of lifesaving operations. Hassan, who lives in Germany, has a genetic disease - junctional epidermolysis bullosa - that leaves his skin as fragile as a butterfly's wings. A piece of his skin was taken, its DNA was repaired in the laboratory and the modified skin grafted back on. After nearly two years, the new skin appears completely normal.
[...] Normally, the different layers of the skin are held together by "anchoring proteins". But the junctional epidermolysis bullosa means Hassan's DNA lacks the instructions for sticking his epidermis (the surface layer) to the dermis (the next one down). There is no cure, and about four in 10 patients do not even reach adolescence.
[...] [A] team of biologists specialising in gene therapy were brought in from the University of Modena and Reggio Emilia, in Italy - and the parents gave approval for them to try an experimental therapy.
In September 2015, a 4 sq cm (0.6 sq inches) patch of skin was taken from an area where the epidermis was still intact. The biopsy was then infected with a customised virus. Viruses are good at getting inside cells, and this one contained the missing instructions for binding the layers of skin together.
The now genetically modified skin cells were grown to make skin grafts totalling 0.85 sq m (9 sq ft). It took three operations over that winter to cover 80% of the child's body in the new skin. Hassan's father said his son had spent months covered in so many bandages he had looked like a mummy. But 21 months later, the skin is functioning normally with no sign of blistering. You can even pinch the once incredibly fragile skin, with no sign of damage.
[...] An analysis of the structure of Hassan's skin, detailed in the journal Nature [DOI: 10.1038/nature24487] [DX], has discovered a group of long-lived stem cells are that constantly renewing his genetically modified skin.
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.
Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures. "I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.
[...] The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated. In November, California scientists tried editing a gene inside someone's body for the first time using a tool called zinc finger nucleases for a man with a metabolic disease. It's like a cut-and-paste operation to place a new gene in a specific spot. Tests of another editing tool called CRISPR to genetically alter human cells in the lab may start next year. "There are a few times in our lives when science astonishes us. This is one of those times," Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.
Previously: Gene Therapy Cure for Sickle-Cell Disease
Gene Therapy to Kill Cancer Moves a Step Closer to Market
U.S. Human Embryo Editing Study Published
FDA Approves a Gene Therapy for the First Time
Gene Editing Without CRISPR -- Private Equity Raises $127 Million
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
FDA Approves Gene Therapy for Non-Hodgkin's Lymphoma
Gene Therapy and Skin Grafting for Junctional Epidermolysis Bullosa
Gene Therapy for Spinal Muscular Atrophy Type 1
Biohackers Disregard FDA Warning on DIY Gene Therapy
CRISPR Used to Epigenetically Treat Diseases in Mice
Gene Therapy Showing Promise for Hemophilia B
Gene Therapy for Retinal Dystrophy Approved by the FDA
CRISPR Treatment for Some Inherited Forms of Lou Gehrig's Disease Tested in Mice