Three people with inherited diseases successfully treated with CRISPR
newscientist.com
newscientist.com
The article does not mention whether that's because putting Chromosome 11 back with CRISPR is harder, or whether for some reason that wouldn't fix the problem.
This isn’t a legacy system, if you want to keep the IT analogy this would be failing over to a different system or replacing an existing solution that doesn’t work with a competitive alternative rather than fixing all your bugs.
Another way to look at it: Would a per-patient fix be possible? Maybe. Is it worth debugging and fixing everyone's crappy DNA to do that? Probably not.
For this specific case it’s also better because lesser coverage might still produce sufficient results since fetal hemoglobin out competes adult hemoglobin.
I think in general CRISPR would be patch over rather than a point fix.
I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.
I'm not saying your attitude is obviously wrong, but there must be a lot of people whose imagination is captured by making tools and the potential of that to change the world. The development of a tool like optogenetics, for instance, makes me feel like amazing things can be accomplished. It's leverage.
Maybe you and I have different definitions of 'platform' company? I define as a way to iterate to get new products, like Regeneron uses their humanized mice to crank-out mAbs, or Genetech/Amgen use recombinant molecular biology to make mAbs, or Vertex uses their in vitro ion channel conductance screening method to make new structure correctors for CFTR...
Was money a problem in general along the way?
I agree that you should give serious pause. Probably the most serious pause of your life, but still. I wouldn't shy away too quickly. When you know, you know.
So you have a much larger pool of genetic and non-genetic diseases that can be cured now. For example if someone had low lung function due to lung scarring, this might be an interesting treatment to look at.
Interesting that they need to kill the remaining bone marrow cells. I wonder what happens when they skip that step. Do the modified cells still reproduce, resulting in a partially effective treatment? Are they targeted by the immune system and eliminated? Are they just out-reproduced by the normal cells and fade into irrelevance?
If the original gene produces a protein that actively causes an issue in the body, versus if the gene produces a broken protein that simply fails to do its job.
In the first case, a partial replacement means that the original active protein continues to cause the disease.
In the second case, a partial replacement means that the repaired protein begins expressing itself.
In either case, there may or may not be a linear correlation between the percentage of fixed proteins and the severity of the disease, and that relationship probably isn't well understood in most disease cases, but a partial replacement is likely much more beneficial in the second case, rather than the first.
That is why these initial test are done on chemo patients, so that we can isolate testing of the method of treatment without needed to know what amount of the original tissue needs to be replaced.
There is still a long complicated road ahead. Not a lot of organs get fully replaced in other procedures.
1. You only give the patient a small number of edited cells. You want those cells to multiply and replace the existing cells. If you don't remove the existing cells, the edited cells aren't going to reproduce very fast because the body has a feedback loop and is saying "we have enough bone marrow cells now, so no need to make more".
2. Host versus graft disease. The old immune system will attack the new immune system cells.
But you’d also gain the donor’s immunity, and if they’ve been vaccinated, the recipient would have that immunity as well.
I've heard it discussed as a possible way to treat autoimmune diseases like MS.
Definition of disease: Resulting from a pathophysiological response to external or internal factors.
Definition of disorder: A disruption of the disease to the normal or regular functions in the body or a part of the body.
Edit: I have it myself (and my kids do too), and I often correct people on this. Since I rather have a disorder than a disease:)
Example: Acutane was once used to treat acne in teenagers. Many/most doctors didnt give it to teenage girls regardless of whether they were sexually active or on the pill. Mistakes happen. That drug would do horrible things to a fetus.
For serious conditions like those in the article, forcing sterilization seems real cruel in an age with safe and plentiful abortion.
Not in the US.
The fix used CRISPR (and a gene engine?) to implement the fix on other people.
It's like version 2 of what human bodies figured out first naturally.
This sounds like something that my dad or other people with COPD/IPF could use. If fetal haemoglobin could grab more oxygen it would benefit people with low lung function. Sort of like Star Trek "Trioxin" they loved to use.
https://www.nytimes.com/2019/10/09/health/mila-makovec-drug....
Please give more details.
Big dental isn’t blocking it we just don’t have a good enough model and yes there isn’t a sufficient financial incentive to develop it really either.
Caries isn’t a big problem these days, it’s not then major cause of tooth problems as people grow older, dental hygiene and more importantly fluoride in water pretty much solved it for those population that were affected.
There is no financial reason to block a caries vaccine as it’s not going to have any impact on the industry, cosmetic and corrective procedures would still be just as in demand and gum disease is by far a bigger factor for tooth loss in adult patients than caries.
Not to mention that quite a lot of the “vaccines” weren’t traditional vaccines but rather replacement therapies where lacto acid producing bacteria would be replaced with strains that cannot produce it but can outcompete the lactobacillus flora in your mouth, these treatments are let’s say problematic since we have had little to no experience in flora replacement therapies and can’t predict or model the outcomes well.
Dr. Jeffrey D. Hillman invented it in the 1970s-1980s at Harvard under NIH grants (https://grantome.com/grant/NIH/R01-DE004529-10). Swab inoculation and you're done. It worked perfectly in animals and humans.
In the 1990s, he founded Oragenics, to try to commercialize it. The FDA gave them the runaround for about 20 years before they finally mysteriously gave up. In 2016, he got a 17-year patent, so I guess it'll be shelved until 2033 at least.
There's something fundamentally wrong with this picture. Why should a simple application of genetic engineering from the 1980s take 50+ years to make it to market? It's either a complete scam or a conspiracy.