Search Results for aav

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…

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…

Domesticating wild organisms– Whether its domesticating teosinte or aurochs, bending wild organisms to our will is always a game-changing event in human history. The domestication of viruses is no exception. We now live in a world where small-pox, a virus that once wiped out entire continents, is now functionally extinct.  Generations of people see iron…

Look, scientists are nuts.  Virologists, on the other hand, are certifiably insane.  As Hedwig would say, virologists make the strangest things seem suddenly routine.  Yesterdays absurd future technology (GENE THERAPY!) is now something as miraculous as curing genetic diseases, and as mundane as an anti-smoking therapy. Todays insane idea? Using mouse and rat parvoviruses to…

GMO Virus for Nicotine Addiction

I used to think gene therapy was an absurd ‘solution’ for HIV/AIDS.  Well, ‘absurd’ is putting it lightly.  I thought gene therapy was a perverted solution– Even if it ‘worked’, it would only be available for the richest people in the richest countries, not the millions and millions and millions of individuals living in poverty…

A long time ago, in a galaxy far far away, ‘visionary’ scientists like Francis Collins thought ‘genetic diseases’ were coming to an end. Because they believed in ‘OGOD’, ‘one gene, one disease’ (lol, *blink*), they were sure identifying ‘disease’ genes would… somehow… give us the ability to cure said diseases. Unfortunately, even ignoring the technical…

Hey, remember a while back when I mentioned how scientists used evolution (random mutation and natural selection) in the lab to ‘perfect’ gene therapy vectors? Theyve just one it again! In a BIG way! Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery. These folks were looking for a way to get man-made viruses…

Say youve got a problem. Maybe you cant figure out how to make a gene therapy vector that cant be detected by pre-existing antibodies that everybody already has. Maybe you are trying to figure out how to make an effective HIV-1 vaccine. You could figure out which portions of AAV or HIV-1 are really important,…

Gene therapy is easy, in theory. Born with a bad gene? No prob!! Just replace it with one that works! We have lots of ways of doing that! I mean, retroviruses plop new genes into our cells all the time! Half of your genome is made of leftover retroviruses! So why dont we just put…