Can someone knowledgable explain why we can't manipulate viruses to patch our genome? I don't know why that doesn't work, I just know enough to realize it would be an enormously useful thing.
I've been speaking with gene therapy researchers, and they do believe that NGLY1 is a good candidate for gene therapy because (1) NGLY1 is a relatively small gene and (2) we're pretty good at transfecting into the critical organs for NGLY1 (like the liver).
Of the treatment options we're researching right now, gene therapy is #3 on my priority list.
One of the reasons I'm bullish on gene therapy and related techniques is that it's one of the few approaches that could cure the many of the rare diseases out there today.
We can and it's a very active area of research, called gene therapy. An early attempt gave a kid cancer, so people are still pretty cautious about it, but things are much improved now, and patients with a variety of diseases have been successfully treated. http://en.wikipedia.org/wiki/Gene_therapy
Another attempt caused a massive immune reaction. That said, the amount of furor over a few deaths in gene therapy trials seems to be irrational: far worse things happen in cancer treatment all the time.
I really wanted gene therapy to be available 20 years ago and even went to grad school to learn how to make it work. It was strongly deemphasized at my school in a preference for classic small-molecule protein inhibitor therapy.
Gene therapy is truly non-trivial. It took me several decades of studies in genomics and protein functional informatics to appreciate how subtle and complex the problem is. I'm hopeful that we can make technology that enables routine personalized gene therapy a common form of therapy, but it is truly not an easy problem. Causing cancer and immune response is only the first set of easily observed (fatal) side effects.
While there are a lot of nuances to this I won't get into, the simple answer is that it's hard to control where the virus inserts into the genome (imagine inserting a random page in the middle of a recipe book).
It's also hard to get the virus to target the cells you need to reach, as they often only target one specific cell type.
The virus doesn't need to insert its sequences into the genome. There are different types of virus, and some do (retroviruses) and others don't.
It can be perfectly fine to have the dna floating around in the nucleus or even the cell body to turn it into a protein. Exceptions abound, of course...
Experimenting on otherwise terminated fetuses would be unquestionably unethical because we'd have to let them age a little, which means their brain would develop, they would gain consciousness and would be likely be very messed up, then have to be "put down". That idea probably won't get much support...
We can and do use monkeys, "Planet of the Apes" is based on this. Attempting to cure alzheimers created smart monkeys, then a bunch of unknown stuff happened and they took over the world.
I'm not going to say impossible, but we just don't need them at this point. Plus since a lot of these genetic experiments would be ultimately affecting something in the brain, it might be near pointless. I don't know, I just don't see it being plausible or necessary or allowed anytime soon.
If we can grow headless bodies, we can just use the organs for transplants, so... that'd be much easier.
I don't know, that's kind of a strange question that I don't feel comfortable even considering. Not many scientists would be comfortable experimenting on headless humans either.
Understanding DNA/RNA and simulating it with computers while using animals to verify the algorithms might be more effective.
Basically science is: predict what will happen, try everything, see what happens, study the results, don't blow yourself up or kill people. Mice and other animals are debatable to harm if necessary, greater good I suppose.
Organ transplants would be the biggest benefit, possibly even full head/brain transplants which would cure almost all conditions. Once we have the technology to reconnect nerves at least.
Yes you can't test what happens to brain tissue ethically, but you can test on every other organ and tissue which would be a huge advantage.
Not necessarily. The population of some countries allows their government to kill (selected) people, but not to experiment on them, so the first doesn't necessarily imply the second.
"modern medicine" isn't perfect, it's good and getting better every day, but we have a long way to go.
Plus, we don't know for sure what modifying our DNA does. It might affect certain things, but it might also have unknown consequences. There is a ton of trial and error in science, and you don't want to have too many errors in your body, else it leads to cancer and other horrible things.
Lots of mice have given their lives to ensure that we and our children live awesome healthy lives. Thank you lab-rats (and other animals), you have suffered for our benefit (though you didn't have much of a choice).
There is a lot of research on this, but "patching up the genome" isn't possible that way.
Gene therapy generally only inserts DNA into a cell (by whatever means possible). Inside the cell, it gets used almost as if it where inside the actual genome.
The problem is reaching enough cells and getting the genes to work well enough...
to sum it you, it's very hard to target the specific location in the genome you want to correct/overwrite. A virus will just insert it's payload where ever it wants, which almost certainly will cause unintended consequences in your genome
