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Related: About this forumA boy with a rare disease gets new skin, thanks to gene-corrected stem cells
From http://www.sciencemag.org/news/2017/11/boy-rare-disease-gets-new-skin-thanks-gene-corrected-stem-cells
A new therapy could restore healthy and protective skin to patients with a rare genetic disease.
iStock.com/Andrey Prokhorov
By Kelly Servick Nov. 8, 2017 , 1:00 PM
A 7-year-old who lost most of his skin to a rare genetic disease has made a dramatic recovery after receiving an experimental gene therapy, researchers announced today. The treatmenta whole-body graft of genetically modified stem cellsis the most ambitious attempt yet to treat a severe form of epidermolysis bullosa (EB), an often-fatal group of conditions that cause skin to blister and tear off at the slightest touch.
The new approach can address only a subset of the genetic mutations that cause EB. But the boys impressive recoveryhes now back in school and is even playing soccercould yield insights that help researchers use stem cells to treat other genetic skin conditions.
It is very unusual that we would see a publication with a single case study anymore, but this one is a little different, says Jakub Tolar, a bone marrow transplant physician at the Masonic Cancer Center, University of Minnesota in Minneapolis who is developing therapies for EB. This is one of these [studies] that can determine where the future of the field is going to go.
EB results from mutations to any of several genes that encode proteins crucial for anchoring the outer layer of skin, the epidermis, to the tissue below. The missing or defective protein can cause skin to slough off from minor damage, creating chronic injuries prone to infection. Some forms of EB can be lethal in infancy, and some predispose patients to an aggressive and deadly skin cancer. The only treatment involves painfully dressing and redressing wounds daily. Bandage costs can approach $100,000 a year, says Peter Marinkovich, a dermatologist at Stanford University in Palo Alto, California, who treats EB patients. Theyre like walking burn victims, he says.
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BlancheSplanchnik
(20,219 posts)I'm interested in genetic syndromes...I've seen vids on YouTube about EB. It's a horrible disease. This is amazing news!
secondwind
(16,903 posts)Cicada
(4,533 posts)We have trained international students who have gone home and started research facilities all over. The number of scientific studies has exploded. We see problems around us but realistically, as Obama has said, this is the best time ever to be born.
BigmanPigman
(51,674 posts)Last edited Sat Nov 25, 2017, 06:15 PM - Edit history (1)
a big No No. I hope the scientists overseas are continuing the work and will shate their results with us. The only science the idiot supports are hair plugs, permanent tans, and weight loss without exercise or diet.
Cicada
(4,533 posts)China and India will overtake us economically and other countries will grow rich and support science as well. Trump will be a mere speed bump on our path to progress.