Gene Therapy
Category archives for Gene Therapy
Using a genetically modified HIV-1 to genetically modify leukemia patients T-cells to teach them how to kill the cancer? YAWN! Thats childs play, at this point. Lets give those GMO viruses a real challenge. Lets get them to fight a real bastard form of leukemia– B-cell Acute Lymphoblastic Leukemia (B-ALL) in adults. In kids, B-ALL…
This is one of the cool ideas not included in the 1800 human gene therapy trials because its still in animal models, not humans: Preclinical Therapy of Disseminated HER-2+ Ovarian and Breast Carcinomas with a HER-2-Retargeted Oncolytic Herpesvirus If Ive said it once, Ive said it 1000 times: ‘Cancer’ is not a monolithic entity. We…
I wish I wish I wish this article was open access! ARG!! Gene therapy clinical trials worldwide to 2012 – an update This article is a great review of gene therapy as a therapy, and how it has evolved from an experiment that killed children to an accepted therapy that is saving and improving lives.…
Hey remember how I told you all scientists are using herpes, the regular-ol much hated cold-sore virus, to treat/cure cancers? The approach has left us with miraculous effects… or no effects at all. I mean, half of the melanoma patients lived A LOT LONGER than they were supposed to, several of them ending up with…
Whoda thought that injecting viruses into peoples hearts would be not only fun, but good for their health! I just wrote about this little guy that can turn regular heart muscle cells into pacemaker cells (in guinea pigs), and here is another cool study hot off the presses: Long-Term Follow-up Assessment of a Phase 1…
When youre trying to cure a genetic disease with a genetically modified virus, you dont have to get a perfect score. You dont have to cure everyone in the trial. You dont even really have to cure them– Just make their lives a little easier, making it so that they can skip a few invasive…
Im seriously, you guys. ANY MEDICAL CONDITION YOU CAN THINK OF: There is a scientist, somewhere, trying to use viruses to treat/cure that condition. The latest: Direct conversion of quiescent cardiomyocytes to pacemaker cells by expression of Tbx18 Lots of people, either through age or genetic defect, need pacemakers. Pacemakers, while functional, come with a…
A lot of folks have been forwarding me this story from the New York Times– In Girl’s Last Hope, Altered Immune Cells Beat Leukemia It is a FANTASTIC story about a little girl who had been sick from leukemia for two years, wasnt responding to conventional therapies, and her parents chose to try alternative medicine. …
Lets play a game. Think of a human disease. Any disease. Viral, bacterial, genetic, acquired, anything. Im pretty sure that no matter what disease you just thought of, there is a scientist, somewhere, trying to use a virus to cure/treat that disease. As I was doing my rounds on PubMed, looking for cool new research,…
So what do you do after you use a genetically modified virus to treat–>functionally cure Hemophilia B? Why, use a genetically modified virus to treat–>hopefully functionally cure Hemophilia A, of course! Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A Though they are both ‘hemophilia’, hemophilia…