Gene Therapy

Tag archives for Gene Therapy

One of my favorite stories is the tale of a GMO virus deployed to treat Hemophilia B: Gene therapy for Hemophilia B … Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of…

You all know me. There are two things I really love: Studying HIV Using viruses for gene therapy One would think I would be over-the-moon about the FDA approving human trials for a gene therapy to stop HIV. HIV! Gene therapy! YAY!! With HIV Cure as the Goal, Gene Therapy Research Expands When this line…

Five kids in the first trial. Then eleven. Now thirty (ultimately 39): Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia Slowly but surely, HIV genetically modified to genetically modify relapsed acute lymphoblastic leukemia (ALL) patients T-cells are prolonging (saving?) kids lives. Ive written about this treatment a couple times before: ‘Dismal prognosis’ with…

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…