Gene editing in healthcare isn’t a novel procedure, but has been seeing fairly recent breakthroughs. The technique has brought us closer to curing paralysis and “butterfly” disease. But in an ambitious first, scientists at Benioff Children’s Hospital have attempted to rewrite DNA in a live patient to cure a rare genetic disorder.
“This is opening up a whole new field of medicine,” said Sandy Macrae, president of Sangamo Therapeutics, which funded the trial. “You can imagine all the diseases that now become possible to treat when you can put in a new copy of the gene, or turn it up or turn it down.”
The experimental patient suffered from Hunter syndrome, which damages organs due to lack of a particular enzyme. Researchers have yet to report on the new method’s success. With only some 12 gene editing trials in progress, the study has a lot to prove but, on the whole, seems promising.
Eric Topol, a geneticist and cardiologist at the Scripps Translational Science Institute, called the new trial “a very important milestone.”
“I’ve been following medicine over 30 years. I’ve never seen anything move at this velocity,”
Thanks to an abundance of brand new technology, gene therapy is getting the boost it deserves. Hopefully it’ll see its patients through to a happy ending.