Four hemophiliac patients successfully treated with gene therapy
Following up on years of preclinical trials, including the curing of hemophiliac mice earlier this year, scientists gave six patients a gene therapy treatment, injecting them with a specially built virus carrying a functioning version of the gene for the defective clotting factor. The virus inserted the gene into liver cells, which proceeded to manufacture the clotting factor, and the patients maintained elevated levels of it for over 6 months. Four of the patients were able to stop receiving injections of clotting factor (the current treatment) altogether.
… welp, here we go! hope we’re careful when we make all these new viruses.
I actually get this. Applications of gene therapy at it’s best *mindblown*