View Full Version : First real gene therapy "cure"?

02-01-17, 01:10

It's a good summary of gene therapy, but this particularly caught my eye.

"The dream of gene therapy is to fix your DNA so you’re not sick anymore—a “cure.” During 2016, Italian scientists at Milan’s San Raffaele Telethon Institute for Gene Therapy reported (https://www.ncbi.nlm.nih.gov/pubmed/27129325) that they had cured 18 children of a rare but terrible immune deficiency disease, ADA-SCID. They removed the children’s bone marrow, added a gene to make the ADA enzyme their bodies lack, and replaced it. Technology Review explained (https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/) how the treatment, now called Strimvelis and owned by Glaxo, took 14 years to develop and test. It was approved in Europe in May of this year."

Apparently, hemophilia could be next.

03-01-17, 10:42
Welcome to the Brave New World

03-01-17, 17:03
I wasn't able to open the link, so I'm sorry if this was mentioned in the article ... but I just read about mice being cured from hemophilia. Which I find pretty amazing: