Gene Therapy

Tag archives for Gene Therapy

WHY IS THIS NOT STANDARD THERAPY YET? Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia I wrote about this last year: ‘Dismal prognosis’ with leukemia? Nothing a GMO virus cant fix. In the previous study, scientists took cytotoxic T-cells from five B-ALL patients, and infected them with…

GMO retroviruses vs Parkinsons

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson’s disease: a dose escalation, open-label, phase 1/2 trial 1. Start with HIV-1s ‘country cousin‘, Equine Infectious Anemia Virus (EIAV). EIAV is a lentivirus, like HIV, so it is a good gene therapy virus. Lentiviruses are a bit more trustworthy than the gamma retroviruses…

More gene therapy for more kinds of blindness!

This therapy isnt perfect, but its a cool start! Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial ‘Choroideremia‘ is a kind of blindness caused by deletion of a gene called REP1. Because REP1 is on the X-chromosome, there is  no ‘second chance gene’ on the other chromosome in men. They…

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…

1800 gene therapy trials

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.…

GMO viruses for coronary artery disease

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…

Building a better GMO virus for Hemophilia B

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…

BWAHAHAHAHA GMO virus to replace pacemakers

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. …