There are a lot of unexplored consequences of potentially editing out a gene as important as PCSK9. Some of the compounds it is making are essential to brain and liver development. They'll have to study that for years.
Also note that in the study linked in the main post, the gene itself is being base edited in the liver, not globally.
By the way, I would like to point out that your ilk have killed a great many people. For decades you have cracked people over the head with the idea that cholesterol is a health outcome and that saturated fat is bad for you. It turns out that cholesterol is a heuristic and that it’s been read wrong to boot. And it turns out saturated fat isn’t just not bad for you, it’s an important part of a healthy diet.
https://www.sciencedirect.com/science/article/pii/S073510972...
Dr. Shawn Baker and Dr. Paul Saladino ate nothing but red meat and saturated animal fat for two years and they had CAC scores of zero. There is testimony that someone’s CAC score went down after cutting out sugar and adding beef tallow.
Can you blame me for resenting the medical establishment?
Getting to your assertions about cholesterol, there is a chasm between "dietary recommendations for cholesterol" and "genetic and therapeutic modification of lipoproteins."
Re: dietary recommendations, which seems to be what you are referring to, I think that the evidence for or against specific dietary recommendations for many types of nutrient intake is hardly compelling. But it's also not an area of my expertise.
Re: therapeutic reduction of cholesterol, the results over decades are totally unambiguous: reducing LDL-cholesterol via statins and PCSK9 inhibition reduces cardiovascular disease and prolongs life. And those benefits are also conferred to people who have lower cholesterol throughout their life due to genetics.
I disagree about LDL. It’s not as simple as LDL. There are different kinds of LDL and some are worse than others as is pointed out in that link, I think. Medications that reduce LDL do other things too and it’s not clear that their effect on LDL is the real vector for their efficacy. And people with elevated LDL sometimes don’t develop the diseases that high LDL supposedly causes. It’s a broken model based on an overly intuitive interpretation of legitimate data.
I read a biochemistry textbook. The same one you guys read. The only time it mentioned ketosis, it was a brief footnote in effect saying “ketosis is a dangerous abnormality and results in diabetic ketoacidosis.” It was literally a sentence or two for the whole metabolic phenomenon of ketosis. A widespread textbook printed in the last 5 years if I recall. That’s when my eyes were opened to the possibility that the doctors have been making oversights. How could you blame a doctor for believing his textbook?
But if you look outside of the mainstream literature, it appears that that high LDL is protective. Also statins appear to cause more problems than they solve ( So has Aseem Malhotra been screaming about for nothing?). While I cannot quote 'studies' ( I do believe that you can find some if you searched ) , there are compelling anecdotes ( like that of Dr. Shawn Baker) which to me proves consensus on a topic need not necessarily mean that the view is correct. Ofcourse people quickly remind you that anecdotes are not data...
Ok I know what you mean, what you mean is that we don't have a formal definition. But I'd like to believe that a perceptive physician does know when people are unhealthy, i.e they observe the person physically, not merely look at numbers. Such physicians are a dying breed though.
If I'm interpreting the article correctly, though, it sounds like in their trials they think they may have edited nearly all the targeted cells.
Using the single-cell sequencing we’ve learned that disease is sometimes driven by a very small sub-population of cells(e.g. cancer treatment resistance). There’s no way of knowing how the organism will respond to a perturbation even in 1% of the cells.
Here is the thing, this is probably moot. The genie is probably already out of the bottle.
I doubt that an average person with CF would want testing on primates to be done in their name.
Justifying your ethical position by name-checking a disability group is kind of gross, especially given that animal testing is not a mechanism which typically results in improved treatment.
I’m sure you’ll find people who don’t feel that way. But I can’t overstate enough how horrible it is to go from being a functional adult with all the joys and sorrows that brings to being a much less functional person in pain every day. That is reality for millions of people. I don’t feel that what GP did was gross. I think it reflects reality for people who suffer on a daily basis, and whose loved ones do.
It's your choice to support that; but there are only about four million non-human primates on the Earth/or, 400,000 great apes.
A good percentage of them are already being tested on.
And I’d have any number of them tested on if it meant progress toward a cure for any number of conditions afflicting human beings. I don’t think that’s “gross.”
Most animal research doesn't have therapeutic benefit as a goal or any prospects of contributing to any therapy. Pretty much none is successful on these terms.
e.g. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1781970/
Even so, animal research as a whole is still justified and gets public funds on these terms, without any evidence of results.
Can you name one therapy, confidently, perhaps even without searching, that has clearly come out of animal testing?
[To answer my own question, the only example that I'm immediately aware of is: thalidomide.]
Why, in any other circumstance, would we want to spend a precious resource in unlimited quantity on something that is expensive, unethical, and doesn't work?
However, to claim that animal research hasn't had a massive positive impact on the advancement of biological knowledge and healthcare is not based in reality.
Animal testing has found hundreds of vaccines against HIV that work in primates, but none of them have worked in humans.
How is that a useful thing to spend time on? You have a model, and it's proven that your model doesn't work: I would expect you to throw away the model.
You could spend those excellent researchers and science budget on anything else, and expect better results.
(I get that all drugs have been tested on animals out of legislative requirement, but I hope it's clear we are talking about using animal models for de novo drug discovery).
For a business-oriented forum, I find it hard to understand why people see this as valuable.
Arguing that someone with a disability should support this because they might benefit, without reason or evidence for that, is an appeal (only) to emotion.
I find it ableist, to use disabled people as an abstract hypothetical grouping in this way, only to support a point that is indefensible any other way.
If you don't want me to have an emotional reaction, don't set up an emotive point.
Your point in the other comment was that you would cause suffering to and sacrifice "hundreds of billions" of primates for a small chance to heal yourself.
That's clearly not balanced. I personally don't think that animal testing as it exists today, and given the complete lack of direct results that it has to show us, is balanced either.
Do you really value yourself, as a human, trillions of times higher than a chimpanzee? I don't and won't respect that. You saying that, out loud, only makes me wonder how much you internally value other humans.
The technique described in the article absolutely could result in not just "improved treatment", but an actual cure. We know what causes CF. We know what causes many other genetic diseases. Right down to the individual bad DNA segment.
And there is absolutely no way those treatments are ever going to be approved without animal testing. Sorry, they just aren't.