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Precision Gene Editing
mpthompson writes "NewScientist.com is reporting that scientists at Sangamo Biosciences have developed a method of editing DNA mutations with unprecedented precision without weaving in potentially harmful foreign genetic material. Different combinations of amino acids are designed to latch on and cut the DNA at exactly the place where the mutated gene lies. This triggers the body's natural repair process which corrects the gene where the DNA was cut. The technique will be used to target diseases caused by single-gene mutations such as combined immune deficiency (X-SCID) - or bubble boy disease - and sickle cell anaemia."
is it just me pr did slashdot add a "daypass" if you watch a commercial to see unreleased stories yet instead of subscribing?
Noone writes jokes in base 13!
The article only mentions cutting the DNA and then "allowing the body's natural repair processes" to do the rest - it seems that this technique could also be useful in inserting genes at precise locations in DNA instead of letting viruses and bacteria insert genetic material wherever they please? I am no genetic engineer, can anyone comment?
So this treatment actually alters the genetic code of a human? So any genetic disease would not get passed down to future generations? How is something like this administered? Our DNA is found in every cell of our body.
--
Fairfax Underground: Fairfax County message board and public records
I would say that's a bit paraniod, and possibly based on some educational time spent watching the sci-fi channel.
Science is full of ethical questions, bio-sciences especially. What we can do we will do ( as a race ), that's a proven fact. It's better to do what we will do in the open, in front of many eyes, instead of being done in a third world country for some wacked out group intent on bringing their own version of reality to pass.
Mod me down with all of your hatred and your journey towards the dark side will be complete!
If sick people can get cured by something like this, we can't afford not to exploit it.
Let's just not forget that there is not such thing as evil knowledge. The way we use it makes good or evil.
With adblock on it was just a matter of clicking a couple times, didn't see the ad. Now I see what I'm missing by not subscribing (hint: nothing).
Sure, we could cause a ton of long-term problems. Keep in mind, though, that this sort of treatment is completely voluntary, and any doctor who doesn't go through the potential risks probably shouldn't be a doctor anyway. That, and the potential for fixing several of those nasty skeletons in the closet known as the human genome is motivation for quite a few, despite the risks.
My other Sig is
Errr...only if you affect the germ cells (sperm&eggs). Otherwise no altered trait can be passed along.
they say diarrhea is hereditary, it runs in the jeans...
A feeling of having made the same mistake before: Deja Foobar
While I see where you are coming from, this process is no different than surgery on a fundamental level. Similar to removing a tumour or cist, it is a proceedure that if done properly can vastly improve the quality of life for the patient. According to the article after the 'cut' is made the body repairs the strand itself, so no insertion of new genes are required.
"and it is among the benefits of science that it equips the future for its duties."
-- Alfred North Whitehead, 1927
I know people who are geneticists, and who work in a lab where they are able to essentially make a mouse to order. You want one that grooms obsessively, here you go! Want one that glows in the dark? You got it. Just because they do it through genetic manipulation rather than breeding doesn't make it any more evil than other means.
What it does do is accelerate our ability to learn about life. Should we take things in measured steps? Absolutely! We should also have been more careful about asbestos, lead based paint, DDT, agent orange and more. But should we ignore these amazing advances? Absolutely not!
The CB App. What's your 20?
Before the first atom bomb was detonated, there were some scientists that thought that the nuclear reaction would spread and ignite the entire atmosphere. Despite their reservations, the tests were done anyway. Screwing up has never been a risk people considered worthy enough to stop a scientific experiment.
Rhymes that keep their secrets will unfold behind the clouds.There upon the rainbow is the answer to a neverending story
>
> Just wondering.
Funny you should ask. I just got this video from Paul Simon.
It's a turn-around jump shot
It's everybody jump start
It's every moderator throws a hero up the crackpipe
Singin' filk is magical and magical is pain, think of the boy in the plastic bubble
I'm a Slashbot with a baboon brain
(And I believe)
These are the days of lasers on a shark's head,
Lasers on a shark's head somewhere,
Staccato signals of constant information,
A loose affilliation of megabytes
And gigabytes and baby...
These are the days of miracle and wonder,
This is a long-distance boast,
The way the duplicate posts appear in slo-mo,
The way we go for first post.
The way we look to a Netcraft BSD troll,
That's dying like a server at NewSci,
These are the days of miracle and wonder
And don't cry baby, don't cry...
I have a feeling that this has to do with homologous recombination, where damage to a certain gene causes the chromosomes to auto-repair themselves by copying the target gene from the "good" chromosome. At least that's my take on why they would mention damaging the DNA to repair it.
I am defenseless. Use your button. Mod me down with all of your hatred.
Great, now the gene splicers have the equivalent of a hex editor, but still have no clue what they are editing. It's like hacking binary code out of one program and inserting into another program and somehow getting it to work.
Until we have a better handle on Gene Expression and how to actually interpret the genetic code we should proceed cautiously.
To quote Dr. J. Craig Venter, Time's Scientist of the year (2000).
"We know far less than one per cent of what will be known about biology, human physiology, and medicine.
My view of biology is 'We dont know shit.' "
If any am being overcautious or am ill-informed please feel free to correct me. I try to live by the motto, "Just because we can do something, doesn't mean we should." This applies to System Administration as much as it does to gene-hacking.
This means the son of the goatse.cx man will not be posing on the Internets.
"short term good"? This has the potential to eradicate several crippling diseases and increase the quality of life of an innumerable number of people. You're going to have to give a better reason against gene therapy than "you're acting as god." You're personal religious opinions are not welcome in a diverse global arena, which is (or ought to be) tailored toward the pursuit of the greater good. You only serve to alienate those of us who may not subscribe to the notion that scientific progress runs counter to moral norms (a concept whose ontological coherence is debatable).
On a related note, this kind of attitude is precisely why scientific progress often stagnates. Irrational fear hinders societal good. Messing up a few times, as cold and calculating as this might sound, may be necessary in order to develop effective medicines and therapies and pinpoint options that do not work. The individuals who sign up for clinical trials are aware of the risks, and those who do should be applauded for their selfless contribution to the good of humanity.
Regardless of your personal beliefs, gene therapy is one of the most promising developments in medicine. It has the potential to revolutionize our perceptions of the human body.
I would say that's a bit paraniod, and possibly based on some educational time spent watching the sci-fi channel.
PFFFT!!! GIVE ME A BREAK! You want the truth!?
I predict the following Prophecy:
Years from now we will have enhanced ourselves to the point where our skulls grow large and our eyes turn black. We no longer need to speak with our tounges, rather our minds. We no longer use sex as a means to reproduce. We have geneticly engineered ourselves to do things even I, Prophetic Truth, can not invision. BUT THERE'S A FLAW! A horrible flaw which can not be fixed by our future selves. A flaw of such consiquence that it will wipe out our species.
The solution?
Time travel, anal probes, and sperm collection.
The Prophecy has already been fulfilled
time is a perception of a being's consciousness
time is your 6th sense, the wierd ones are 7+
While the gp was really just being flamebait, there is a significant element of danger in genetic engineering.
Example: biobricks are really advancing, and you can already get custom genes made for a price that anyone can afford. Lets say that biobricks advance to the point where it's relatively trivial to make a gene that produces proteins that create Sarin, for example (or perhaps a different nerve agent with a longer life). You insert the gene into a common strain of phytoplankton found all over the globe, along with another gene to help give it a competitive advantage against its wild relatives. You then release it into the ocean (preferably in multiple points to speed up the process), and wait. The discovery of the source of the gas would likely be too late; almost everything with a nervous system would end up dead as the wind carries the gas across continents. A bioengineered apocalypse, in short, produced by someone with a bit of bioengineering knowledge and a couple thousand dollars investment.
sed "s/SJW.*$/... never mind. I was about to say something stupid, and also, I'm a troglodyte./Ig"
Just stay away from the brown X-SCID man.
Forgive me for not believing in your esoteric views of this "God" character nobody has any proof of, but I feel genetic manipulation is going to be one of the few things that allow us (the human race) to continue existing.
As time goes on, we defeat simple diseases such as the bubonic plague, then upgrade to tougher ones like smallpox. We're now at the point where the only communicable diseases that are seriously fatal are biologically engineered bacteria, and viruses. On top of that, we've still got Cancer to worry about, which is kicking our asses.
While it may be cheaper to produce drugs for everyone alive and distribute them to everyone, no company in their right minds would do this. But if we could figure out genetically how to teach our immune systems to deal with cancer, and certain foreign invaders, we could save millions simply by changing our children's genes.
I think the biggest paranoia attributed to genetic engineering is the fear of change; just because we know how something works now, and we assume that it'll continue working the same way into the future, we give up the notion that we can change things for the better or for the worse. Yes, we are foulable creatures, but at the same time, we now know how to clean up our mistakes. It's far past time we take our fates into our own hands. Why use medicines that can screw up other things in our bodies when we can simply prevent the problem from occuring naturally?
"Victory means exit strategy, and it's important for the President to explain to us what the exit strategy is." G.W.Bush
I think there is a natural equilibrium between nature and gene mutations. When the hand of man starts changing one side of the equation, can the consequences on the otherside be foreseen? For example, who is to say that some form of cancer today won't mutate to something 1,000 years from now that will save humanity from some enviormental change?
Rosco: "If brains were gunpowder, Enos couldn't blow his nose."
http://www.biologynews.net/archives/2005/04/05/res earchers_pioneer_new_gene_therapy_technique_using_ natural_repair_process.html
like the 'bubble boy' defect mentioned in the article, we often know the specific bit of code that causes the problem.
n odeficiency
"IL-7 signalling pathway
Most cases of SCID are derived from mutations in the c chain in the receptors for interleukins IL-2, IL-4, IL-7, IL-9 and IL-15. These interleukins and their receptors form part of the IL-7 signalling pathway.
The IL-2 receptor (IL-2R) gene is located on the X chromosome and mutation of this gene causes X-linked SCID.
Janus kinase-3 (JAK3) is an enzyme that mediates transduction of the c signal. Mutation of its gene also causes SCID."
http://en.wikipedia.org/wiki/Severe_combined_immu
Just lable the experiments as labratories making weapons of mass destruction. LOL. Bomb. Invade. Elect pro-western government. Move on to next country.
But seriously. With genetic engineering, do you think that a third world country will work on curing cancer when they can work on building roads or opening hospitals?
The true threat is some western scientists move shop to a third world country. But we can track them down and arrest them for violating international law. Plus, the possibility of being punished in the third world country, and not the USA should be a huge detterant. We don't even need a fair trial.
Rosco: "If brains were gunpowder, Enos couldn't blow his nose."
You exist in a dimension of space and time and you think its all coincidence..The universe has no purpose, its just here.
TFA noted that the zinc fingers cue in on two sets of 6 base pairs to find the site that needs correction. Assuming randomness in the base-pair sequences, this 12 base-pair key will bind with approximately 1 out of every 16.8 million (actually 1 out of every 8.4 million due to complementarity of the base pairs). Given that the human genome has about 3.2 billion base pairs, this means that the modifier will match in 381 positions more or less.
Thus, this method will fix the error in one place and introduce an error in 380 other locations. The key needs more than 16 base pairs to be statistically assured of homing in on a unique mutation (depending on the statistics of DNA, it may need more or less).
Two wrongs don't make a right, but three lefts do.
I'm allowed to think whatever I want, and as there is no proof in any general direction, I simply don't think about it. Besides, if "God" didn't want us to manipulate our genes, it would stop us.
I have not read the article, but repair processes can be "error prone". That is, the mechanisms cells use to repair DNA often involve high error rates.
/.ers may not appreciate is that typically, it is VERY, repeat VERY hard to get chemcial reaction specificity of anywhere close to 1e9 for reactions invovling DNA.
The human genome is 3e9 BP long (roughly..not counting indels, the unsequenced centromeres, etc etc)
So the chemical process of identifying the one single mutated basepair has to have a chemical specificity of >>1e9, because there are >>1e6 cells that are exsposed. That is, lets say you feed the reagent to a person. Millions of cells, each with 1e9 bp, are expsosed. Say the process has an error rate of 1e10 - many, many cells will have incorrect repairs done
This is just like error rates in, say, reading data from a harddrive: the larger the file, the lower the error rte has to be
What
I will rtfa,
Really, emacs is a whole lot of stuff that just happens to have text editing functionality along with it, so why not genes?
I note that the process involves removing blood from the body, running the process on cells purified from teh blood, and then reinjecting the cells back intothe patient.
I believe, but am not an expert in this field, that the simple process of removing cells from the body is in and of itself, highly mutagenic.
There is *no way* the cells could be checked before reinjection.
In any event, it is an interesting piece of science, but a LONG way from clinical practice - stay tuned for the update in 2020.
I think you meant the biology news link to the same research
Your original has some extra characters and can't be used.
-- Tigger warning: This post may contain tiggers! --
Pharmacorp executive: "Let's see now, we can sell them a one-time treatment that cures them for the rest of their lives, OR we can charge them $1000/month for drugs to maintain their current status for the rest of their lives... well, obviously we'll choose the method that is best for the patient's well being, our profits be damned! I mean, it's not like we have a board of directors that will sack us if our revenues don't increase every quarter!"
I've abandoned my search for truth; now I'm just looking for some useful delusions.
Bubonic plague isn't "defeated"... FYI.
sed "s/SJW.*$/... never mind. I was about to say something stupid, and also, I'm a troglodyte./Ig"
in 500 years, and between then and now millions of people suffer painful deaths to avoid changing something that might be helpful in the case of your hypothetical event?
Anyway, there is the whole somatic vs. germ line thing, if genetic engineering is limited to somatic cells, changes won't be passed on to children (unless we start reproducing via mitosis).
Maybe something you don't understand is that diseases, cancerous cells and the like are an evolution of life themselves. Something such as a cure for cancer is not only hypocritical in the sense that it is essentially killing a form of life, the fact is that it is an expression of natural population control.
Being stuck in a master-slave mentality of good and bad makes it remarkably simple for points of view like yours to seem reasonable when in fact they are intrinsically hypocritical. Just because something hurts or kills doesn't make it bad and just because something feels good or prolongs life doesn't make it good.
[obscure Seinfeld reference] The Mooks.
germ line changes.
If a person has a terminal disease, somatic changes may or may not help, but they aren't likely to cause more damage than the disease.
And by the time they have a terminal (or even chronic) disease, you can get a pretty good idea how "the organism will express it's genes".
Treating disease in somatic cells is a much different issue from creating new lines of plants/animals/humans via changing germ line cells--at least in organisms that reproduce sexually.
I am getting real tired seeing all this great research, only to realize that treatments are the same tired treatments at the beginning of the century.
NONE OF THIS RESEARCH has changed people lives.
Stop with the fairy tales. People have not and will not get any new cures to any existing health issues.
GE is the technological revolution to shame them all, and will have massive impact on our society.
That said, I'm all for it, and will be first in line for gills.
"A language that doesn't affect the way you think about programming, is not worth knowing" - Alan Perlis
because viruses never alter DNA
"It has always been this way and it won't change, god bless the fucked up USA" The Briefs
our brains allow us to control nature, not just exist in it. We can do whatever want, but we should be responsible when it comes to genetics because it puts our entire species at risk. If a person wants to design their genes let them, but there must be some rules and standards.
Thats why we don't depend on that industry. Buy your drugs in India, take a vacation and come back with your genes fixed.
"Just because something hurts or kills doesn't make it bad and just because something feels good or prolongs life doesn't make it good."
If you don't believe that human life is intrinsically valuable, and if you don't think that our cognizance and rationality are unique characteristics, then there is something fundamentally flawed about your point of view. Human life is uniquely valuable and we ought to make efforts to improve it. READ: It is not okay to acquiesce supinely while individuals needlessly die, especially when techniques are available to prevent such a thing from happening. Prolonging and improving human life is good. Ending it prematurely is bad. Simple? Sure. Deontologically sound? Definitely.
But it does give us the ability to create the equivalent of patch files for bad/defective genes when a good/functional version of the gene is available.
There are many genetic diseases where the mistake in the DNA is well characterized, and it is very clear exactly what difference between the normal version of the gene and the defective version causes the disease, even if we don't have a full understanding of what the hell gene does; we just know to a high degree of certainty that a particular error causes a particular phenotype.
This new technology, if it lives up to the hype it's given here, could mean we can fix these kinds of diseases.
Yikes!!!!
Actually, we have a whole number of 'seriously fatal', excessively natural, diseases left. AIDS, Flu, Ebola, Malaria, and TB come to mind, plus the various drug-resistant streps, West Nile.
Genetic manipulation might allow us to finally treat diseases by some method other than mining other organisms for specialized toxins, then hoping the disease (which reproduces on the seconds to minutes time scale) doesn't become resistant too quickly.
the more accurate the calculations became, the more the concepts tended to vanish into thin air. R. S. Mulliken
I don't understand your arguement that cancer is a form of life. It is simply your own cells that have become defective. Curing yourself of cancer is like having your appendix removed (except that it's harder to achieve). It's not a form of life. Your immune system actively does this every day.
Cancer isn't an evolution of life either - life has spent billions of years evolving to a point where it is really good at preventing cancer. A car with smashed-in bumpers and its engine stuck on isn't a new type of car, it's just a damaged one.
Sure, cancer does contribute to population control, but if you're worried about that then maybe you should be campaigning against soap and fresh drinking water. Those make more of a difference than cancer treatments ever will.
Yeah, THAT makes sense.
...the only communicable diseases that are seriously fatal are biologically engineered bacteria, and viruses.
:)
As opposed to what, just-kidding-fatal?
My children can finally be bred as Valids!!!
guess it's a matter of perspective.
aren't cancer cells defective in that they are effectively immortal? They divide endlessly, choking off pertinent cells in an organ or system? to that end, they are a form of life... just an entropic form. thus the defect in cancerous cells is the greed with which they use resources and disrupt essential functionality.
i'd go as far to say that understanding and being able to control some of the processes in cancerous cells will lead us to significantly increase our life spans.
going of on a tangent: are cancers cells prone to the same replication errors that normal cells are prone to over a long enough time span?
un burrito me trampeó.
The way to look at cancer is not as a thing that is invading the body, but as a colony of things. They grow, divide, evolve and die. That is why it is generally fatal. The first chemo treatments will often kill >90% of cancer, but what survives is the cells that have mutated to be resistant. So the doctors prescribe a different chemo. Once again the susceptible cells die, but the colony goes on.
There are basically only three ways you can permanently beat cancer:
1/ Sometimes you can cut it out - and get it all.
2/ Sometimes you can beat it down to the point where the immune system can beat it.
3/ Sometimes the cancerous mutation involves a weakness that makes a treatment 100% lethal to the cancer.
But those are rare, mostly you are buying time not a cure.
Back to the tangent, yes they are susceptible to replication errors, but in a colony those provide diversity for evolution to select from. That is one of the things that make cancer so nasty.
I'm guessing that wasn't on their radar screen...
See, I guess mostly fear from change are not because changes are bad. Changes are usually good - those who doesn't kill us, make us stronger, and we learn a bunch of new stuff even if things don't work out as we hoped for.
Problem is in inrationality, emotionality hidden by... rationality. If everything is done in colobrative way (like open source), I would believe that process. And such change in state of mind of the human is started - but it comes slowly. People don't trust each other, people don't trust other nation, people better hang on on the stupid believes which leads them nowhere than risk with trusting to others.
Because if someone fails them, they hearts are broken, they don't want to trust anyone. They better off with single-minded, narrow-wided point of view, because it allows them to live peacefully.
For example, someone says coorporations are out here for money. NOT exactly. I guess they are out there for satisfy their greed to money and power. Why? Because companies are usually headed by someone for whom money and power is everything in his life. Must be a very misguided soul, if you ask me. And that is what I FEAR most. I fear human being who claims to have reasonable interests, yet, it he is laying to himself to make himself happy.
Went a little offtopic, but nevermind...
user@ubuntubox:~$ stfu This server is going down for shutdown NOW!
Hello Greg Bear, are you reading this? We need a trilogy. Maybe Darwin's TV. For /.ers -> for reference read Darwin's Radio and Darwin's Children. Much fun and profit in them there genes.
Too lazy to create a sig...
Um....I don't mean to be a jerk, because cancer is a horrible horrible thing, but how is it kicking our asses? True, we haven't found a cure for all of it, but we can sure as hell get rid of a lot of it. And don't forget that our races birth rate still far surpasses our death rate. Now, the bubonic plague, that wiped out 2/3 of Europe, that is significant.
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In fact that's exactly what the article says: "This triggers the body's natural repair process, called homologous recombination, which corrects the gene where the DNA was cut, The researchers provided the cells with a copy of the correct gene as a template."
If they were to concentrate this work on Myotonic Muscular Dystrophy, they could likely achieve a success very quickly. It is caused by an unstable CTG sequence of DNA that expands in length when replicated. The progression of the disease is characterized by the number of expansions. Since it is an unstable sequence and of little use, simply cutting it out of all DNA should "cure" the disease. I put the "cure" in quotes because reversing the damage is likely not possible, but it could at least eliminate it from future generations and stop the progression.
Selective breeding in the past did not create the equivalent of a "critical mass". One or two relatively minor mutations across may be tens or hundreds of specimens, then later, more interbreedings to select the characteristics. All the while, nature and time being the moderator allowing the weak, damaged, or DANGEROUS to fail or be isolated in the necessarily small group. Instead, a hurried implementation of mass genetic change could conceivably create a critical mass, such as planting a gene corn throughout the world, and though maybe technically inferior over a long time, but strong enough to survive in the ecosystem and disrupt native species, even though the erosive nature of time and nature would eventually win by selection.
Does ten small, hairless dogs have a chance to propagate in the wilderness?
Does a million? A big difference.
I simply cannot agree that old style breeding == new style en-masse genetic manipulation.
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
FYODOR D. URNOV1, JEFFREY C. MILLER1, YA-LI LEE1, CHRISTIAN M. BEAUSEJOUR1, JEREMY M. ROCK1, SHELDON AUGUSTUS1, ANDREW C. JAMIESON1, MATTHEW H. PORTEUS2, PHILIP D. GREGORY1 & MICHAEL C. HOLMES1
1 Sangamo BioSciences, Inc. Pt. Richmond Tech Center 501, Canal Blvd, Suite A100 Richmond, California 94804, USA
2 Department of Pediatrics and Department of Biochemistry, University of Texas Southwestern Medical Center, 5323 Harry Hines Blvd, Dallas, Texas 75390, USA
Correspondence should be addressed to M.C.H. (mholmes@sangamo.com) or M.H.P. (matthew.porteus@UTSouthwestern.edu); requests for materials should be addressed to M.C.H.
Permanent modification of the human genome in vivo is impractical owing to the low frequency of homologous recombination in human cells, a fact that hampers biomedical research and progress towards safe and effective gene therapy. Here we report a general solution using two fundamental biological processes: DNA recognition by C2H2 zinc-finger proteins and homology-directed repair of DNA double-strand breaks. Zinc-finger proteins engineered to recognize a unique chromosomal site can be fused to a nuclease domain, and a double-strand break induced by the resulting zinc-finger nuclease can create specific sequence alterations by stimulating homologous recombination between the chromosome and an extrachromosomal DNA donor. We show that zinc-finger nucleases designed against an X-linked severe combined immune deficiency (SCID) mutation in the IL2Rbold italic gamma gene yielded more than 18% gene-modified human cells without selection. Remarkably, about 7% of the cells acquired the desired genetic modification on both X chromosomes, with cell genotype accurately reflected at the messenger RNA and protein levels. We observe comparably high frequencies in human T cells, raising the possibility of strategies based on zinc-finger nucleases for the treatment of disease.
Most human monogenic disorders remain difficult to treat because therapeutic transgenes do not undergo homologous recombination (HR) into the mutated locus1, 2, and gene addition by virus-driven random integration remains a challenge owing to transgene silencing, improper activity or misintegration3, 4. Furthermore, targeted alteration of DNA sequence in vivo--in principle, a powerful basic research technique for studying genome function--in mammals requires sophisticated targeting vectors and drug-based selection1, 2, which limits the use of this approach5-7.
The C2H2 zinc-finger, originally discovered in Xenopus8, is the most common DNA binding motif in all metazoa9. Each finger recognizes 3-4 base pairs of DNA via a single alpha-helix10, 11, and several fingers can be linked in tandem to recognize a broad spectrum of DNA sequences with high specificity12-14. Engineered zinc-finger protein (ZFP)-based DNA binding domains with novel specificities have been extensively applied in vivo to target various effector domains12, 15. Work from the Chandrasegaran laboratory has shown that a ZFP can be coupled to the nonspecific DNA cleavage domain of the Type IIS restriction enzyme, FokI, to produce a zinc-finger nuclease (ZFN)16, which then cuts the DNA sequence determined by the ZFP16, 17. An important specificity mechanism derives from the requirement that two ZFNs bind the same locus, in a precise orientation and spacing relative to each other, to create a double-strand break (DSB; Fig. 1a)17. One mechanism by which eukaryotic cells heal DSBs is homology-directed repair (Fig. 1b)18-20, which transfers information missing at the break from a homologous DNA molecule (Fig. 1b). Work from the Jasin laboratory21, followed by that of others22, 23, demonstrated that the endonuclease I-SceI can potentiate HR into loci previously engineered to contain its own recognition site, and the Carroll24, 25 and Baltimore26 laboratories have shown that a ZFN-invoked DSB increases the rate of HR in model systems.
Figure
In this case, the Zinc Finger Nuclease is simply used to cut the defective DNA to initiate the homologous recombination process. If you had bothered to read the abstract you would understand that they are also providing an "an extrachromosomal DNA donor" (I would suspect on a plasmid) as the source for the corrected DNA sequence.
So this process *need not* be error prone. Of course they are obviously looking at a number of cells after the fact to determine the fraction of cells in which the process was successful. If one did this with stem cells (which seems to be where they are going) and put them back into the body then one would indeed be able to correct SCID or sickle cell anemia. Diseases that are present in adults in specific cell types such as cystic fibrosis or muscular dystrophy are going to be a bit trickier.
Speaking as a type-I diabetic, it would be nice to be cured, but not at the expense of becoming the carrier for some god awful flu mutation that kills 100 million people. When you can't even quantify the risk, it's usually a good idea to pour more research into what you are doing.
Yeah we can treat it, but at what cost? I know more people who have cancer than I'd like to have known; practically all have either died of the cancer being non-responsive to chemo, or die because the chemo reduced their immune response so severely that an ordinary sinus infection or the flu killed them.
Once again, Slashdot obscures the meaning of "Our". If you look at deathrates of the United States, Cancer is up at the top of the list, next to heart disease. Heart disease can be dealt with by simply eating better, no need to fix our genes there, but cancer, we can simply give people a highly toxic drug cocktail, and tell them goodluck.
"Victory means exit strategy, and it's important for the President to explain to us what the exit strategy is." G.W.Bush
To have a technique that could possily cure people and then not use it for "religious" or for whatever fear is to my mind immoral - we may as well be living in the dark ages. Obviously the technique needs work still, and there are always risks with any medical procedure. Provided the patient knows fully those risks and agrees I don't see a problem, particuarly when no harm to anyone else is being done. The technique isn't introducing any alien material into a human. AND in any case we are already mutated by dioxin and radiation and any number of chemicals eg PCB's etc. in the environment. So how is this technique going to change the face of being human? And to those who have made fun of the illness, I suggest that you fully investigate the illness and the suffering it causes thousands of people every day. There are {at current tally} at least 85 variations of Primary Immune Deficiency - X-linked SCID being only one of them. The technique mentioned will only cure one variation anyway. Not the other 84. Still it is worth pursuing as more knowlege is another link in the chain eliminated. Check out; http://www.jmfworld.com/ http://www.pia.org.uk/