I dedicated a significant fraction of my life to the idea that gene therapy would become a viable medical treatment. It seems unlikely to really become a high-impact treatment.

Being able to change DNA in cells is one thing; actually being able to show your treatment does what it's supposed to and didn't have negative effects is hard. There are a limited number of diseases where gene therapy should work great, but there are a wide range of others where it won't, until solve multiple grand-challenge class problems.

Would you elaborate on what makes a disease a good fit and these grand-challenge class problems?

A disease is a good fit if you can just inject the therapy into a localized region (say, the eye, or an organ) and the treatment works for a reasonably long period of time (months+). Typically another requirement is that the target is a defective gene where the phenotype can be repaired through addition of a "corrected" form of the gene, without the defective gene needing to be removed. This is the case in X-linked retinitis pigmentosa, where the genetic cause of the disease has been understood for some time, it's relatively simple underlying mechanism (so we think), and you can deliver the medicine to the retina with periodic injections.

Treating a disease where you have to remove an inserted retrovirus from a large number of freely circulating or "hidden" cells (which is the case in HIV) is far more challenging- you need a way to recognize the cells of interest, access all of them, and get 100% transversion. All without causing negative side effects.

We learned early on that one of the difficulties in eliminating HIV is that it hides in the nervous system and can reemerge at any time. This article show promise in that it can reduce HIV viral load during active shedding. I think it is less likely that it could ever eliminate HIV entirely (i.e. cure)

which is why press releases like this aren't really accurate and create false expectations

I do agree, but that is just the good news. In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form, but there is not a force on heaven or earth that is going to stop parents from trying to give their children every advantage.

> In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form

Why is this the right thing to do? What are you signifying by putting "right" in quotes?

I am reminded of the Hyperion Cantos from Dan Simmons, where most of the human race has declared that nanotech modification of the human organism/germline is a cardinal sin. Meanwhile there is another segment of humans who decided that maybe it wasn't, and they're off colonizing deep space by remaking themselves into a wild range of body types.

In fact if we don't stop dirtying up the planet, we may feel a lot more pressure to adapt our bodies to an environment whose rate of change we can't keep up with. In any event I am sure there will be a segment of humanity that does want to preserve human life in a currently recognizable form, and they should be able to choose that for themselves. That should not stop those of us who want to vary it wildly from doing our thing too!

There are a number of ethical concerns when it comes to these kinds of changes, of course. There is the world of The Windup Girl, where competing agricorps target each other's crops with tailored viruses to wipe them (or their consumers) out. So we will need to figure out how to rebalance our societies in the light of this vast new power. Yet it looks like it may also be the start of a whole new stage in treating human diseases. Imagine a world where mass-produced medicines are mostly replaced by taking some host cells, gene sequencing the target diseases, then programming and re-injecting the cells to eliminate them. I'm not well-versed enough in this stuff to know how far-fetched that is, of course. It sounds like something out of Star Trek. But it's hard not to be optimistic about what it can enable.

> to do the "right" thing

"right" according to whom? nature itself doesn't believe that

Despite academic/political/military/banking/corporate alignment against fundamental morals, "right" appears to be in agreement with nature/natural selection. The States had a more "right" society founded om constitutional and civil rights that was a big part of its climb from an English colony to the world power in less than 150 years after the constitution was written.

The reason people probably have strong convictions about right or wrong is most likely because of nature, not despite it. Groups that had this genetic trait built tribes, societies that had greater success in the long term.

In the short term, individuals, or even groups of individuals, can be successful by acting amorally, but this comes as a cost to their group's long term success--the cancer analogy.

Right now there is limited competition between groups in the world. Most of what we should be competing against is the coming extinction event when something else out there gets a whiff of all the artificial elctromagnetic radiation. Probably the reason we hear so little in the cosmos is because it is not a very competitive strategy.

> academic/political/military/banking/corporate alignment against fundamental morals

"fundamental morals"? What are those and where the hell do you get them from?

> The reason people probably have strong convictions about right or wrong is most likely because of nature, not despite it. Groups that had this genetic trait built tribes, societies that had greater success in the long term.

Actually, couldn't agree more. But people do have _different_ convictions about right or wrong.

Re fundamental morals:

I would wager >95% of people agree on these. I've never met someone who didn't have an understanding of fundamental morals, except for a few people whom I consider to be a psychopaths.

I really would bet that most of us dont need an education to know that killing children for pleasure is wrong and disgusting. However, we might be split on whether spanking your children as punoshment is wrong. I think people understand the difference.

> I really would bet that most of us dont need an education to know that killing children for pleasure is wrong and disgusting.

Can I remind you that abortion is legal and funded by the state in most of the first world?

It's not that I have a personal opinion on the matter (I find it too complex to develop a definite opinion), but it does come pretty close to what you're describing.

It's not. An egg moments After fertilization experiencing suffering requires quite a bit of inagination, while we all can agree a child can suffer. Where we draw the line between that is obviously somewhat up for debate (and no one in their right mind is saying we should kill born babies for pleasure).

You seem to be using subjective experiences and the ability to experience them as base for your moral system. Is it the case? Why?

I dont believe for a second that let's say you did something you knew to be evil, such as kill a child for convenience, and were hypothetically asked about this in an after life: "why did you do it, you knew it was evil," that you'd be able to say honestly that you didn't actually know that.

As far as I am aware anything is possible, but suffering and happiness seem to be real.

> we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form

Why do you think that preserving human life in currently recignoziable form is something all of us are optimistic for?

For me personally, this option seems like a nightmare - and I truly hope for a transhumanist future made of humans who have very advantage. Being smart, fit and (relatively) healthy is awesome, which I'm lucky to know from experience. I wish other people would be able to experience it, and then some.

>although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form

That race is already over. Technology over the last century has changed our realities so much that we can never hope to go back.

Seems too good to be true

To be fair, they probably said the same about penicillin when it was discovered.

And in a way penicillin is too good to be true, now that bacteria are widely resistant to it and many other antibiotics.

Penicillin probably saved at least tens of millions of lives, and other antibiotics even more. Yes, some strains of bacteria are developing resistance, but even today antibiotics are saving many many lives every day.

Can we crispr resistant bacteria to be susceptible to penicillin/antibiotics again?

Interesting idea. So you would first infect voluntarily with a custom virus that targets the bacteria by using CRISPR to remove the DNA strand of the resistance, then simply use the usual antibiotics to kill it.

At that point couldn't you just make a custom virus which kills the bacteria, so it happens in one step?

Doesn't erase the benefit it has had up to this point.

The millions saved haven't been eroded if some bacteria now are developing resistance.

Fortunately in the late 19th century so did the prospect of Moon exploration and arrogated human knowledge in our pockets. But it's pretty standard now

Moon exploration isn't standard, but I get your point.

Aggregated* not arrogated.

Evidence?

That was the plan from the start, no? I was pretty disappointed to learn that most drug research is essentially mass testing all kinds of molecular agents that happen to show some beneficial effect for a problem at hand. It seems just too simple a method.

That is rapidly changing to a more targeted approach.

However, don't downplay the importance of the shotgun approach. In the broadest sense, it gave us Viagra for ED (common reported side effect in an unrelated trial), Aleve (intended to be a hangover treatment you'd take the night before), ...

With computer and analytical modeling, it's a much easier process to identify a problem and reverse engineer a treatment.