In a First, US Doctors Use Crispr Tool to Treat Patient with Genetic Disorder

The lack of ability to process out ammonia seems pretty debilitating, so that seems like it was a good candidate.

Jesse Gelsinger is a hero, and should be remembered as such.

That all said, we should glean as much science as possible from the postmortem to understand and prevent whatever misunderstandings resulted in the death, going forward.

I watched the video. I am not a biologist.

I could have sworn at one point he said that it would be inserted at "known, and safe" locations. Not "random"... ?

First (which I don't really want to concentrate on) is that's why this is an experiment. People did stupid things (and sometimes died because of them) for entire human history and some hundreds of years later are remembered as "great scientists". Trying to monopolize research and experimentation around academia and "certified professionals" (certified by academia, that is) is a most despicable thing I can imagine. I see that this opinion is basically a point of conflict of two different political views, so there isn't much that can be said to (quickly) persuade someone, who does not feel like this in the first place, but I strongly feel that if people can be "free" in any sense at all, they really should be free to do whatever the fuck they want to themselves. If they wanna die, let them die. If they wanna do drugs, let them do drugs. If they wanna perform stupidly dangerous experiments over themselves, let them do that. If they wanna give somebody else a consent to perform a stupidly dangerous experiment over them — it's their prerogative.

Second is much less philosophical and much more grim. This probably depends on your environment quite a lot (country/place, level of income, connections), so take this as my subjective experience. The problem is that dealing with certified medical professionals performing FDA (or whatever) approved procedures over yourself sometimes feels pretty much as dangerous, if not more. You often have no idea what they are really doing to you, and it's sometimes blatantly obvious they don't really understand it either. Often it is not clear if the treatment (which might be totally safe and correct in some cases!) is adequate to your case, not much of analysis is performed and every little bit you have to do along the way costs quite a lot of money and/or prevents you from living normal life, leaving you quite miserable overall. And the more minor the thing you are trying to fix is, the worse it is. And the worst of it, it is pretty clear that your doctors don't really care. They are relatively safe (legally) if they are working by the book, so they are working by the book. And "the book" often happens to be a result of bureaucratic machine, not common sense. And, after all, they have seen (maybe just 30 minutes ago) people in much worse condition than yourself, so unless you are really dying, what's even the trouble? And all of it even when we ignore the fact, that "the book" and the status quo of the field changes all the time, much of recognized medical research lacks the quality you wish it'd had and accepted views today sometimes are vastly different than 20 years ago. And let's be honest: 20 years of "experience" is nothing, when we are talking about high-stake decision making on the frontiers of the scientific research (and whatever affects your health and well-being always feels like "high-stake").

- He just possibly infected his whole digestive system. Not just small intestine, but stomach as well. Furthermore, AAV can potentially exhibit transcytosis through epithelial layers, suggesting that it's possible the virus infected more than just his digestive system.

- He did not determine an appropriate dose, and so he likely infected with a HUGE genetic payload. Overexpression with AAV can kill infected cells, which means this man is risking his digestive lining.

- Neither the promoter nor the encoded protein itself are human, potentially risking (possibly severe) autoimmune reaction.

- There are few/no long-term studies on effects of AAV integration and expression in humans. There is indeed evidence that AAV increases risk of cancer, almost certainly in a dose- dependent manner.

- AAVs could integrate randomly into your genome in low frequencies (0.1% to 1%), meaning that they could just by chance disrupt a gene you really need to not get cancer.

Let me rephrase, to make this more explicit: why shouldn't people be allowed to commit probabilistic suicide by non-rigorous experimental protocol, if they want? People do things that could kill them in home machine shops and home chemistry labs all the time, just for fun.

Note that I completely understand why people shouldn't be advertising that they do this, any more than people should be trying to talk other people into building nuclear reactors as a hobby.

I'm more saying that, if the point is just do something for your own sake—with a chance of improving your life, and another chance of killing you—and nobody else is involved or will be involved—then what has anyone else got to do with it?

I trust you (haven't checked biohackers much myself), but as with any field of knowledge/research, the opposition you suggest has nothing to do with credentials and everything to do with intellectual honesty, integrity of the researcher.

I mean, let's take programming since it's HN. Say you compare two programs. You could follow the scientific method (hypothesis -> experiment(s) -> conclusion -> new hypothesis -> etc) like any decent engineer or scientist would... or you could just randomly "feel" which is better "in your opinion" — basically being a charlatan with your own topic.

Unless you're doing marketing, you should be selfishly attracted to the truth — which is really better / faster / etc — because you need the tool for yourself, not to sell it to someone else. I'll never understand charlatanism in research, I won't; but even toy benchmarks for lulz or journaling can be made scientific, easily. It's just a logical framework, which I'd think any self-proclaimed 'reasearcher' knows in and out...

This is isn't to say that you can control all variables in real-world experiments — biological and social research are the hardest in that regard. Sometimes you gotta take a leap because you won't ever be able to "prove" anything beforehand, it's only retrospectively in statistics that you'll know (at the level of metastudies). We've taken many such a bets with public health, family health, our own; whenever the `estimated risks / perceived benefit` ratio makes sense.

I do agree it's wrong to categorize beyond these facts, e.g. credentials of the author of a good idea, or lack of "proof" thereof, have little to no meaning historically — can't stop an idea/solution whose time has come. I mean, Js is the most popular language with Python, let that sink in for a minute. (lol)