That is great news and my congratulations to the parents. Hopefully it's something that we can repeat many times in the future.
Baby is healed with first personalized gene-editing treatment
441–450 of 570 posts
Re: Baby is healed with first personalized gene-editing treatment
#442This is incredible work. Its jaw dropping to learn that something like this is possible at all. Sometimes I wish I could work for a company whose products make a meaningful positive contribution to the work. Do companies like this have a need for SWEs? Are there opportunities for a backend SWE without any background in hardware or biology?
I run a small software team at a small biotech working on diseases with small patient populations, and the answer is yes x 1000. The issue is that in drug companies, software isn't the product, so SWEs will never make as much money nor be as much of a priority as in tech-proper.
There are two categories of software we need help with:
1. Salesforce for science. We don't have big data in terms of volume; we have big data in terms of heterogeneity. Tons of small data sets that need context to be interpreted, including measuring uncertainty. This software, often called an eLN or LIMS, is offered by expensive vendors who each have their custom, locked-in implementations. Every organization needs customization on top of this that can be developed and change with the changing direction of the bench scientists.
2. Informatics tools. Much of the heavier computational tools (bioinformatics, molecular dynamics, stats) were developed by academic labs, who don't have the training or incentives to create sustainable software. Alternatively, they are made by vendors who write software on short-term contracts, so they don't have expertise in house. Our mass spec vendor told us to put their analysis servers on our Citrix so employees could access it. Citrix! If you can convince those vendor to hire you and rewrite their software, please do.
Despite cool tools like alphafold making headlines, the software needs in drug development are more mundane. We need people who are excited to sit down with bench scientists and help them figure out how very normal tools can be applied to their work.
Re: Baby is healed with first personalized gene-editing treatment
#443Do you think someday soon we will have the equivalent of an LLM coding assistant, but for gene editing?
Definitely not soon, there's a lot of conditions at the moment for this to be applicable (from what I gather, I'm no expert, just a HN commenter), and well before wholesale gene editing is a thing there will be the legal aspect. I mean the precursor to gene editing is selective breeding, which on humans quickly leads to eugenics.
Don’t know why so much fear over eugenics. You either resign to waiting millions of years for evolution to do its thing, or we just take matters into our own hands at some point and evolve faster.
Re: Baby is healed with first personalized gene-editing treatment
#444Earlier quoted context omitted.
What an insanely callous and shallow take. I don’t even know where to start. You’re trying to claim the moral high ground by complaining that a privileged child didn’t suffer and die? Do you even understand the point of reducing poverty?
An absolutely terrible and tone-deaf way to phrase that thought, but the fact of the matter is that most of the world (you and I included, in all likelihood) will not get access to this sort of thing in our lifetimes. Not because modern medicine won't have been there yet, but because our lives (and those of our children) are simply seen as being worth significantly less than a rich person's desire to become richer. H…
What are you talking about? Since the 1800s people have been shipping vaccines to "most of the world"
Everyone could afford to "eat healthy" and get exercise if governments and social planners put in a modicum of effort. Unfortunately they aren't directly incentives to do so.
Framing either of these things as a wealth issue ignores both how wealthy even the poorest in the world are and the systems responsible for the problem. For everything else there's health insurance, yet another horribly mismanaged system.
Re: Baby is healed with first personalized gene-editing treatment
#445Earlier quoted context omitted.
Yep, this effort is the culmination of 50 years of research. Could be the last harrah of the NIH with the amount of cuts we've had and the scientists who are taking jobs in other countries.
Unfortunately a staggering amount of research in other countries is largely funded by the NIH/USA.
The reason for this is very pragmatic actually. We don’t have enough researchers of a particular specialty in one country alone. When you get that specialized the air is very rare.
By pooling our funding / effort we can create a larger body of collaborators to solve problems faster and better.
It could be that the organizations are funding wild stuff that isn’t salient. I’ll concede that.
However, in basic sciences there are so few specialists it is important to share resources. The funding is worse than ever (hello 2006!), and that trend is unlikely to reverse for a while.
Source: I worked in bioenergetics for 10y, my collaborators were from Hungary, Chile, Canada, Israel, Italy, and more! At a major conference on mito energetics they all fit in one big lecture hall (100ish?)
Re: Baby is healed with first personalized gene-editing treatment
#446Earlier quoted context omitted.
One other fun part of gene editing in vivo is that we don't actually use GACU (T in DNA). It turns out that if you use Pseudouridine (Ψ) instead of uridine (U) then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous. But , the RNA -> Protein equipment will just make protiens it without any problems. Which, yeah, that's a miraculous discovery. And it was wel…
That also sounds like a recipe for a terrifying virus
I don't thnk the body has a way of making mRNA with pseudouridine.
Re: Baby is healed with first personalized gene-editing treatment
#447If someone in the year 2050 was to pick out the most important news article from 2025, I won't be surprised if they choose this one. For those who don't understand this stuff - we are now capable of editing some of a body's DNA in ways that predictably change their attributes. The baby's liver now has different (and better) DNA than the rest of its body. We still are struggling in most cases with how to deliver the D…
the usual next questions will be: - how further can we push this to make the best, most optimized human? - what are moral implication of this? - what are the side effects / downsides?
There's no "most optimized human". We are already that, perfected in millions of years. What could really happen is the split between multiple sub-species. For example, it makes perfect sense to do the optimization for orbital station dwellers or Mars colonists or underwater dwellers.
Re: Baby is healed with first personalized gene-editing treatment
#448Earlier quoted context omitted.
What does EA means here ?
"Effective Altruism", something I find myself aligned with but not to the extremes taken by others.
Note that these people often condescendingly refer to themselves as "rationalists," as if they've unlocked some higher level of intellectual enlightenment which the rest of us are incapable of achieving.
In reality, they're simply lay people who synthesize a lot of garbage they find on the Internet into overly verbose pseudo-intellectual blog posts filled with both the factual inaccuracies of their source material and new factual inaccuracies that they invent from whole cloth.
Re: Baby is healed with first personalized gene-editing treatment
#449Earlier quoted context omitted.
> on the grounds that vaccines are something you only need to take once so the toxicity issue could be dodged But we didn't take these vaccines once. We took many of them. Am I to understand a known side effect is liver toxicity for multiple doses?
I guess the issue is not about taking the medicine once vs twice, but rather « a few times » vs « daily »
It sure would be nice to see the data on length of time between doses to prevent toxicity. The fact they deleted all of that data sure is suspicious and incredibly worrying.
Re: Baby is healed with first personalized gene-editing treatment
#450Earlier quoted context omitted.
Bear in mind that they intentionally choose something that was soluble - ie the easiest thing possible. So it's doesn't mean everything is now solvable. For example it's no coincidence this is a liver disease as basically almost everything you inject in the bloodstream ends up concentrating in the liver by default - if you needed to target another organ with your LNP it would be much harder. Most of the time people a…
> that was soluble solvable
soluble has two meanings.
- able to dissolve in solvent
- able to be solved.