New Gene Editing Tool Could Fix Most Harmful DNA Mutations (theguardian.com) 30
Scientists have raised fresh hopes for treating people with genetic disorders by inventing a powerful new molecular tool that, in principle, can correct the vast majority of mutations that cause human genetic diseases. From a report: The procedure, named "prime editing," can mend about 89% of the 75,000 or so harmful mutations known to mangle the human genome and lead to conditions such as cystic fibrosis, sickle cell anaemia, and a nerve-destroying illness called Tay-Sachs disease. The landmark work opens the door to a new era of genome editing, but scientists caution that more research is needed before it can be safely used in humans. Beyond proving its safety, another major hurdle is how to deliver the molecular machinery to cells that need it in sufficient amounts to treat a disorder.
"This first report of prime editing is the beginning rather than the end of a longstanding aspiration in the life sciences to be able to make any DNA change in any position of a living cell or organism, including potentially human patients with genetic diseases," said David Liu at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. The ability to rewrite the genetic code is one of the most striking scientific advances of recent years. The most common approach, known as Crispr-Cas9, has been likened to "molecular scissors" which home in on a particular DNA sequence and then cut it in two. The procedure allows scientists to disable specific genes and even correct harmful mutations by providing cells with fresh strands of DNA with which to repair the cut.
"This first report of prime editing is the beginning rather than the end of a longstanding aspiration in the life sciences to be able to make any DNA change in any position of a living cell or organism, including potentially human patients with genetic diseases," said David Liu at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. The ability to rewrite the genetic code is one of the most striking scientific advances of recent years. The most common approach, known as Crispr-Cas9, has been likened to "molecular scissors" which home in on a particular DNA sequence and then cut it in two. The procedure allows scientists to disable specific genes and even correct harmful mutations by providing cells with fresh strands of DNA with which to repair the cut.
If only... (Score:2, Troll)
...we could just make all DNA perfect. Just a big planet full of perfect-DNA-having people. They'd all certainly respect each other.
[/sarcasm]
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Sure, just don't give any of the guys a small penis, unlike Gattaca where everybody got a small one, and everything is gonna be ok
The lack of aggressive behavior from the guy with the Napoleon Complex will do a lot to make the world a better place to live in
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The flip side is that people may be less likely to raise up to correct mass injustice.
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More technical sources (Score:5, Informative)
Technical description with illustrations [broadinstitute.org]
Paywalled original paper [nature.com]
This is a BFD (Score:1)
If you have a disease that is due to a genetic defect, this is good news. Please, no sneers. There's still a long row to hoe for clinical applications, but it is a very positive finding.
How do you get the fix to all the body's cells? (Score:2)
Assuming you have a technique that can repair DNA in some relatively small number of cells, how do you propagate the fix throughout the body, or even just throughout an affected organ system?
Anyone know the thinking here?
sorry wrong number (haha) (Score:2)
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Around 37 trillion cells
And more than half of them aren't even human cells
https://www.bbc.com/news/healt... [bbc.com]
And to answer your question , it'd probably work best in the extremely young. But who knows, cells replicate themselves all the time. Get in on some of that action , maybe it's possible.
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Basically, this is a benefit for cases where you are affecting the genetics of a single cell, not an adult
We have a lot of experience trying to change the DNA of a living person, and unlike Blade Runner, where there was some dramatic negative effect, the changes simply fail to effect enough cells to make a change
For adults, understanding their DNA and designing treatments based on small-protein supplements (pterostilbene, resveratrol, finetis, etc...) would be a lot more effective
Re: How do you get the fix to all the body's cells (Score:2)
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In some cases genetically-based diseases are the effects of a failure of cells to produce certain materials the body needs. A cure could consist of inserting just enough cells to produce an adequate amount of the missing materials.
Progress is being made on another front, that of generating whole new organs from a single cell. The procedure becomes:
Extract cells from a defective organ.
Fix a cell.
Grow a new organ from the fixed cell.
Implant the newly grown organ.
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ie most of the organ systems should test as ready for new upgrades in normal populations.
Too much damage passed down generation after generation and the organ system wont be as ready.
Test a lot before upgrading?
Re: How do you get the fix to all the body's cells (Score:2)
Viruses know how to get into cells, so itâ(TM)s pretty obvious that you can try to smuggle it in using a virus capsule (typically Adeno-associated virus seems to work best) or a forget viruses you can use lipid-based structures such as a nanoliposome or well there are a whole bunch of methods â" not going to list them all here. They arenâ(TM)t very great though .. each approach is great in one thing but suffers a drawback in another .. many labs including mine are working furiously on this pr
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With technology like that you could cure cancer! (Score:2)
Just let me know when (Score:2)
Is it retroactive? (Score:2)
Dumb question. Let's assume this new process does reverse genetic mutations. Would someone with Down's syndrome "revert" to what we would consider normal features, intelligence, etc?
Or is that a different type of gene manipulation?
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Let's ask the leading expert on this topic: Mr. Charlie Gordon. ...
Nevermind.
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Bad liver? Grow a new one and ensure its perfect for that person?
Bad back? Ensure whats repaired is perfect for that person?
Industrial exposure? Replace the entire lung with perfect new lung?
Intelligence? What decade? A person in their 20's makes the investment? Takes a decade to "learn" with their new ability? Super IQ ready in their 30's?
Body totally changed by some condition? How much can be swapped out per human?
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Something like Down Syndrome (which affects whole chromosomes) might be cured in utero if it was detected early enough, and certain effects like heart problems could be treated later, but reversing the full effects in an adult seems extremely unlikely.
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Would someone with Down's syndrome "revert" to what we would consider normal features, intelligence, etc?
Only if that person was a newly fertilized ovum a few minutes ago.
Whoo hoo! (Score:2)
Monocultures are awesome and just what we need!
I just want (Score:2)
A third set of teeth in my fifties..
OK... (Score:1)
Unintended consequences (Score:2)
Some of those unfavorable genetic mutations have a reason. Sickle cell anemia, for example, is related to a gene that confers some malaria resistance:
https://www.sciencedaily.com/r... [sciencedaily.com]
Of course sickle cell itself is caused by inheritance of two copies of the offending gene. Are we going to attempt to completely eliminate sickle cell from the population by preventing gametes from transferring the gene to offspring during mating? Will we limit proliferation of the gene through sexual reproduction to one sex
Herrenmenschen? (Score:2)