All of us have to get real and demand immediate speed-up of LLMs, Gene Therapy, etc. Absolutely nothing else matters.
Politics could do with an injection of LLM action. Climate change is arguably the most urgent issue.
Gene therapy restores hearing to children with inherited deafness
211–220 of 231 posts
Re: Gene therapy restores hearing to children with inherited deafness
#212Earlier quoted context omitted.
I thought you can’t just inject genes and then the body will start replicate it. Does it need periodic injections?
If the DNA is in the cell's nucleus, the DNA will be utilized to produce whatever gene is encoded in the DNA. But if the DNA is just floating around on its own it will not survive a cell division event and the material would need to be periodically reinjected to keep working. However, if the injected DNA is part of a full chromosome, it will be replicated when the cell divides - and will be permanent as long as the c…
This is actually not totally correct. AAV’s form episomes, which are circular DNA structures that are basically extra chromosomes. The fact that AAV’s don’t randomly insert themselves into DNA makes them safer for clinical uses like this.
Re: Gene therapy restores hearing to children with inherited deafness
#213Earlier quoted context omitted.
Taking it seriously for a moment, that still seems outlandish even in a sci-fi scenario. The amount of changes that would be necessary to the body for a human to achieve autonomous powered flight would be substantial. Given our overall density, we would need enormous wings and musculature. Consider that the heaviest flying birds alive today weigh maybe 40lbs and have a wingspan of 8ft or so. If we naively extrapolate…
"...are you suggesting coconuts migrate?" Seriously, lesser-schooled folks don't realize the extent to which evolution took tiny variations over an extremely long time to achieve physical flight. The way it arose differently in pterosaurs, birds and bats is fascinating. I think younger children are capable of understanding more about genetics than we often suppose. More of these concepts should be taught at younger a…
I have a bachelors in bioengineering, I’m not a “lesser schooled individual”. Just because evolution took a long time doesn’t mean we have to, you can run millions of simulations virtually, something that nature has to do over millions of years. Are the simulations going to be perfect? Probably not but there’s a lot that can be gleaned through simulations combined with observations in the real world.
Re: Gene therapy restores hearing to children with inherited deafness
#214Earlier quoted context omitted.
If the DNA is in the cell's nucleus, the DNA will be utilized to produce whatever gene is encoded in the DNA. But if the DNA is just floating around on its own it will not survive a cell division event and the material would need to be periodically reinjected to keep working. However, if the injected DNA is part of a full chromosome, it will be replicated when the cell divides - and will be permanent as long as the c…
This is actually not totally correct. AAV’s form episomes, which are circular DNA structures that are basically extra chromosomes. The fact that AAV’s don’t randomly insert themselves into DNA makes them safer for clinical uses like this. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2519600/
And that’s why replies are helpful in these kind of explanations. My over-edits became wrong, and then an hour later, unfixable. bglazer is correct.
AAVs, unlike Lentiviruses, do NOT integrate.
Re: Gene therapy restores hearing to children with inherited deafness
#215Earlier quoted context omitted.
This assumes that the fundamental nature of human beings can't be changed, which is the presupposition that genetic engineering denies. The entire point of genetic engineering is to try to engineer people at the cellular level. A fanciful example would be modifying human metabolism to be more similar to those of birds that consume most of their calories in the form of simple sugars. Humans can't eat a diet of 100% su…
Are you saying you could see sugar being somehow folded into the many different proteins that make up the vast number of tissues in our bodies. Or are you saying our tissues could be constructed out of sugar crystals instead?
Proteins can be synthesized from sugar as a carbon source. We know this because there are bacteria that can survive and reproduce with no nutrients in their environment other than glucose.
In fact you don’t even need sugar. Carbon dioxide and water alone are sufficient. Plants produce their entire biomass using nothing but carbon dioxide, water, sunlight and trace elements from soil.
Amino acids and proteins don’t occur without life. Living creatures have the ability to synthesize all amino acids and proteins from simpler compounds.
Re: Gene therapy restores hearing to children with inherited deafness
#216Earlier quoted context omitted.
Discoveries like this should be shared with the world - not gatekept by rent-seeking venture capitalists. Wouldn’t you rather be Jonas Salk than Scrooge McDuck? https://en.wikipedia.org/wiki/Jonas_Salk > Salk was immediately hailed as a "miracle worker" when the vaccine's success was first made public in April 1955, and chose to not patent the vaccine or seek any profit from it in order to maximize its global distrib…
Rent-seeking has a technical definition; earning money from things you invented does not meet it. While Salk is obviously an inspiring example, a wise society should happily incentivize the development of useful technology.
There are incentives to developing medical technology beyond the opportunity to become obscenely wealthy. If you do so, you are necessarily depriving some subset of patients that need the treatment.
Honestly, I find it pretty unimaginative (and a bit disturbing) that the only “incentive” you can think of for treating sick children is the ability to monetize the treatment - it’s a virtuous endeavor in and of itself. I must say, though, it’s hardly surprising to meet someone with such a worldview on a VC-focused forum.
Edit:
> Rent-seeking is the act of growing one's existing wealth by manipulating the social or political environment without creating new wealth.
https://en.wikipedia.org/wiki/Rent-seeking
This aligns neatly with my comment - gatekeeping a medical treatment behind artificially high costs to enrich yourself is the very definition of rent-seeking. I think you’re digging yourself in to a pretty deep philosophical hole here, solely out of some need to nerdsnipe people to reassure yourself of your own intellect.
Re: Gene therapy restores hearing to children with inherited deafness
#217Earlier quoted context omitted.
Intellectual monopolies disincentivize development and incentivize rent seeking. Here, rent seeking is legal action and lobbying intended to protect and extend monopolies, instead of advancing science. Other than economic logic, we have natural experiments to back this up: i.e. the steam engine patent granted to Watt and Boulton in 1772 and its chilling effect on engine duty improvement (and the explosion of progress…
I agree with all of this. The example of the patent on steam engines is fascinating, thank you! It's certainly true government granted monopolies are far from the ideal incentive structure, with many downsides. (And lobbying for EG patent extensions likely IS rent-seeking). Advance market commitments and other systems likely have better characteristics. I was rather objecting to the misuse of a technical term, and th…
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Re: Gene therapy restores hearing to children with inherited deafness
#218Earlier quoted context omitted.
This is actually not totally correct. AAV’s form episomes, which are circular DNA structures that are basically extra chromosomes. The fact that AAV’s don’t randomly insert themselves into DNA makes them safer for clinical uses like this. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2519600/
And that’s why replies are helpful in these kind of explanations. My over-edits became wrong, and then an hour later, unfixable. bglazer is correct. AAVs, unlike Lentiviruses, do NOT integrate.
The reason I know this is I made the same mistake in a comment on a similar story a few weeks ago!
Re: Gene therapy restores hearing to children with inherited deafness
#219Earlier quoted context omitted.
Intellectual monopolies disincentivize development and incentivize rent seeking. Here, rent seeking is legal action and lobbying intended to protect and extend monopolies, instead of advancing science. Other than economic logic, we have natural experiments to back this up: i.e. the steam engine patent granted to Watt and Boulton in 1772 and its chilling effect on engine duty improvement (and the explosion of progress…
I agree with all of this. The example of the patent on steam engines is fascinating, thank you! It's certainly true government granted monopolies are far from the ideal incentive structure, with many downsides. (And lobbying for EG patent extensions likely IS rent-seeking). Advance market commitments and other systems likely have better characteristics. I was rather objecting to the misuse of a technical term, and th…
Ah gotcha, agreed.
Re: Gene therapy restores hearing to children with inherited deafness
#220Earlier quoted context omitted.
Each gene therapy is a unique "medicine". There is little economy of scale, at least for the hardest part.
That's due to a lack of theory and useful abstractions in the tools used to modify genes. We're so early on in the development of that field that if this were computers, we'd still be assembling devices by arranging logic gates by hand. Custom cosmetic gene therapy is a great way to incentivize the better tools.
No, not really.
Genes are far more complex than inserting a sequence into a delivery mechanism.