Suppose one had a genetic condition such as sickle-cell anemia or hemophilia. Is it theoretically possible to construct a virus which changes the DNA in cells to eliminate these diseases without destroying the cells themselves? What if you only had to change the DNA in one type of cell? I guess the main issue is I don’t think viruses can reproduce without destroying a cell, but maybe there is a way.
This is exactly the method used in current research on gene therapy.
Safe and effective gene therapy has been something of a holy grail in biotech and pharmaceutical circles for decades.
I worked for a long time at a company dedicated to a type of gene therapy that didn’t use any kind of viruses. This should theoretically eliminate a lot of potential safety problems. Unfortunately the company ran into technical obstacles that eventually proved unsolvable and our efforts in that direction were eventually abandoned.
I don’t know any companies that currently have any large scale research efforts devoted to gene therapy.
Ah, interesting. I never really knew what gene therapy was. Great answers.
I know that Adenovirus used to play a key role in this research because they are large enough to carry human genes, but during some trials (in 1999, it’s mentioned on Colibri’s site) someone actually died from a huge dose. So now the safer adeno-associated virus (AAV) is being used, there are many serotypes that bind to various cell receptors and can be used to theoretically carry the DNA into the cell and insert it into our genome. It’s a wonderful area of research, but probably a ways off from being to the point where we can say “hey lets inject ____ gene into someone in this specific cell type at this exact location and have it express exactly this amount of protein”. I can’t wait until we can though 