Gene Editing Offers Hope For Treating Duchenne Muscular Dystrophy (nytimes.com) 48
schwit1 writes with news that scientists have used a new gene-editing technique called CRISPR to treat mice with defective dystrophin genes. This is the first time that such a method has successfully treated a genetic disease inside a living mammal. The Times reports: "Three research groups, working independently of one another, reported in the journal Science that they had used the Crispr-Cas9 technique to treat mice with a defective dystrophin gene. Each group loaded the DNA-cutting system onto a virus that infected the mice's muscle cells, and excised from the gene a defective stretch of DNA known as an exon. Without the defective exon, the muscle cells made a shortened dystrophin protein that was nonetheless functional, giving all of the mice more strength."
Hope is good (Score:3)
2)Remember the religious furor over stem cell research? The same God didn't make it mantra faded quickly when the technology began to pan out. Tur
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No, the FDA SHOULD spend the time and make sure any treatment doesn't cause harm. Remember thalidomide.
Many molecular biologists seem to have this hubris that they completely understand the mechanics of DNA and genes. They are so specialized, they do not know what their tinkering may do to an organism down the road and long after they have been published or even dead. The scientific method, although quite rational, can take many many years to ferret out issues that have never even been thought of - and like
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No, but most of them in the USA *are* anti-science and do believe the earth is 6500 years old.
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The number is growing: evangelicals' numbers are on the rise, as people from other, more "mainstream" denominations convert. Also, one of those YECs is running for President and is very popular.
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And you base that on what? Let me guess, Answers in Genesis website? In case you haven't noticed, they aren't primarily in the USA, and furthermore, most well known "bible has all answers" types aren't even in the USA. For example, remember the banana guy? Try New Zealand.
Anyways the continent I suspect would be most opposed to this would be Europe. Why? Well nobody else (besides Africa) has outright bans on known safe genetic modification of plant and livestock.
In fact the USA has been leading the way in t
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I base it on being an American and looking at the people around me. This place is full of fundies. Just go to any "non-denominational" Christian megachurch and listen to what they preach about. And keep in mind, these churches are *growing*, while the "mainstream" liberal sects are literally dying out.
Secondly, look at who we elect. The GOP controls most state governments plus Congress. One of its more popular candidates is a young-earth Creationist (and many of the others are likely Creationists too)
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I base it on being an American and looking at the people around me. This place is full of fundies. Just go to any "non-denominational" Christian megachurch and listen to what they preach about.
So? According to this only 18% of them are young earth creationists:
http://ncse.com/rncse/30/3/ame... [ncse.com]
Secondly, look at who we elect. The GOP controls most state governments plus Congress. One of its more popular candidates is a young-earth Creationist (and many of the others are likely Creationists too).
Just for perspective, in the last election in France, 28% of the vote typically goes to the official Fascist party, (Front National, source [wikipedia.org]) which is quite higher than the confirmed number of young earth creationists in the US. Denmark also pushes similar numbers, and about 10 other European countries sit somewhere around 18%. That's JUST the fascist party, and doesn't include any other right leaning groups
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In fact Earth was known not to be flat before there were christians.
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How about actually BEING a patient?
It's one thing to "merely watch". It's quite another to be the one with his ass on the line. As such, I agree with those that advise actually following the scientific method and obeying the usual regulatory controls.
Validating these cures requires scientific rigor, not wishful thinking.
Only 1 in 10 "miracle cures" actually make the cut. None of this is simple, easy, or certain.
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If it is a germline modification, or if somebody can't keep their genetic engineering virus from hopping around, a lot more caution will be warranted b
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The practical application of this form of treatment will (as all three papers do) use systemic, viral delivery, meaning all cells will be affected. You can modulate the probability a little by selecting a virus with affinity for particular tissues, but there will still be germline modifications. If you're really worried about that, you make vasectomy a required co-treatment.
It's still a long way from useful. All three papers report similar results: 2-5% changes in the DNA pool; 40-60% changes in the mRNA
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I agree this technique is still a long way from useful, though.
Re:Hope can be horrible (Score:3)
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Many molecular biologists seem to have this hubris that they completely understand the mechanics of DNA and genes. They are so specialized, they do not know what their tinkering may do to an organism down the road and long after they have been published or even dead.
As a molecular biologist, most of us know that we don't completely understand the mechanics of DNA and genes. We do, however, know a lot more than the average person, and we know a lot about the dystrophin gene, as we've been studying it for a long time. We also know that even if we do screw something up, it's unlikely that it'll cause something worse than DMD already is. Furthermore, this cure - and indeed, any gene therapy cure that could go to clinical trials at the moment - is not passed on in the germ
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> And lastly, the Bible is a few thousand years old - not a thousand - and was written by stone age goat herders who thought the world was flat.
That more accurately describes your own ancestors. The Jews not so much.
They had a sophisticated society with universal literacy when your ancestors were living in caves and eating other.
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It's a complicate two-sided situation. Remember thalidomide... sure, remember it. Also remember that while a medicine is in testing, not yet available to the public real people are suffering and even dying. Those people that die waiting for a cure... they aren't coming back... EVER!
Beyond all that the longer and more beurocratic you make the process the more expensive it is. No, I'm not arguing money over lives but that money could be used to save more lives. The cost of testing is either passed on to pa
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Christians do not have a problem with science or medicine. We have a problem with killing babies and using their body parts. There are now ways to produce stem cells without killing babies and so stem cell research is no longer an issue. The only Christians that have a problem with at least some medical technology (such as blood transfusions) are the Jehovah's Witnesses. They are a small minority Christian denomination.
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3) Cancer.
(Gene editing has a history of being imprecise, keep in mind to treat a genetic disease this also means delivering to each affected cell in the body.)
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(Gene editing has a history of being imprecise, keep in mind to treat a genetic disease this also means delivering to each affected cell in the body.)
Not true. Most genetic diseases don't require treating every affected cell, and in some cases not even a majority need to be treated. DMD only really affects the muscles, so no non-muscle cells need to be changed. Furthermore, since this is a gene editing strategy, they could probably afford to only (or primarily) target the muscle stem cells.
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If you have a disease that WILL kill you in 5 years, the risk that the cure might give you a disease that might kill you in 6 years probably seems worth while.
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A lot of cancer is secondary, induced by the treatments that were used to deal with the previous cancer. Current cancer treatments are quite brutal and tend to do a lot of collateral damage.
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2) Religious nutcases will lobby for a ban on this. Because God doesn't want you messing with genetics.... he only wants your donations.
Why would this bother religious nutcases in particular? The last I saw, the anti-GMO crowd did not look particularly religious. (Well, not fundamentalist Christian, anyway. They might be new-age Gaia worshipers.)
Doping (Score:2)
It will be only months until it will be used for some athlete's "Muscular Dystrophy", then we'll know if it's safe.
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Actually, no.
It helps sick people by removing faulty part of the DNA. If you aren't sick, in best case, it'll make your scores worse.
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It's a real pity, there would probably be some very interesting stuff there if the dataset were available. At least the Purity of Sport remains unsullied, so there's that.
Quirks and Quarks (Score:3, Informative)
Quirks and Quarks did a podcast [www.cbc.ca] very recently about this technology and its application on a particular strain of MD. This work was done (by Dr. Ronald Cohn from the Hospital for Sick Children in Toronto) on living cells however, not live mammals. The podcast does go into a high level and easily understood description of how the technology works. Fascinating stuff.
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Radiolab also did a netcast [radiolab.org] on this.
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I would have like to have up-voted the link to more information, both podcasts are interesting.
There are many lectures on YouTube requiring varying degrees of expertise to understand but this one seems comprehensible to anyone with a STEM background and I recommend you take a look. Jennifer Doudna. The CRISPR-Cas 9 Genome Engineering Revolution, UC Berkeley Events channel (one of many excellent primary sources on YouTube)
https://www.youtube.com/watch?... [youtube.com]
I appreciate that many people with an interest will us
Let's approach this with caution, NOT phobia... (Score:4, Interesting)
I know there are concerns around human genetic manipulation, but there are a lot of people suffering in its absence. I'd be willing to take the risk on a therapy like this if I were suffering from a debilitating or fatal genetic illness. Furthermore, I am ready to shoulder my portion of the societal and ethical risks entailed by others testing it.
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It would depend on the ability of the virus to infect them. Since viruses tend to destroy the host cells when they replicate (which would make the gene splicing useless since the fixed cell is now dead), I would assume that this particular virus would be engineered to not replicate, preventing it from spreading through the body.
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I'm not so sure the concern is even valid in this case given that the germline is already compromised.
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The summary indicates that this virus infects muscle tissue. I don't know how selective a virus can be, so I don't know how effectively you could select one that specifically targeted or refrained from targeting germ cells or their progenitors.
It seems that making a modification that can be carried down the female line would be trickier, since egg cells are all produced long before puberty begins. You'd need to infect those cells directly, I'd think, and I imagine any such therapy would lag well behind the
Adverse selection against hippies (Score:2)
CRISPR is about to bring us an avalanche of genetic engineering on the human genome to attack a variety of gene-related diseases. This is our chance to eliminate the anti- science moment the way Darwin intended, by selecting out people who oppose GMO technology. Good riddance!
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Yes. Because releasing a bunch of little frankensteins out into the wild is the same as keeping them confined to a single human host. You are ever so intellectually superior for being willing to completely ignore these distinctions, or consider the trustworthiness of exactly WHO is using a particular technology. Your scientific hubris is simply fabulous.
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When it comes to fighting something like genetic disease, we need more scientific hubris. We need more of the old sense of adventure that once made us first in applying science to real-world problems. If we want society to fund more research, public or private, we need to make more use of the results research provides.