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Gene Therapy Cures "Bubble Boy"
bofh31337 writes "NewScientist is reporting that Welsh boy Rhys Evans has been cured of the fatal severe combined immunodeficiency ("bubble boy") disease. The medical team, lead by Adrian Thrasher, was able to take the stem cells that give rise to immune cells from his bone marrow and add a normal copy of the gene to the stem cell using a retro virus. Seven months after treatment, Rhys was cured."
I thought he was cured by scientology!
VISA's cure for celebrity shoplifters...
tcd004
Sadly there is no cure for the people who got sick after seeing the movie "Bubble Boy"
http://www.kubuntu.org/
...science can answer the Moors/Moops riddle which has plauged mankind since the dark ages!
The potential power of stem cell research is clearly evident in this case. My question, not to troll, is whether this type of research could have been possible/allowed in the US. AFAIK, the laws in the states allow a restrictive amount of stem cell research -- would this have been enough for similar treatment here?
The Guardian, and Yahoo
My other sig is also a
Paul Simon has announced a full recall of his "Graceland" CD...
Cache Rules Everything Around Me
Truly awesome.
Indeed this is good news however genetic manipulation is not something to be taken lightly. While at the moment this child has been cured what are the side effects of such a treatment later in life? What is to say that this won't spawn some new disease that affects the rest of use.
I fear the use of technology that we do not understand.
Apologies if I sound alarmist.
Well, so then, if the stem cells are placed next to a Shakey's Pizza, they would become another Shakey's Pizza! And you'd have your own Shakey's Pizza where you didn't have to charge yourself to eat!
www.nih.gov/news/stemcell/index.htm has the latest information about what's taking place in the U.S. in regards to stem cell research. It's a great resource for anyone wanting to learn more about this amazing new science.
The biggest problem with gene therapy is that long term expression of the target gene has been difficult to achieve. The inserted sequence, depending on the gene carrier, may or may not be inserted in to host genome. Actual insertion into the host genome is undesirable because of possible malignant transformation (insertion of the target sequence disrupts the function of a tumor supressor protein, or turns on a pro-tumor protein, etc.). Existing as a genetic sequence outside of the genome proper has also failed to achieve more than temporary expression of the desired protein.
This article describes a technique to increase the effiency of the transfer of a therapeutic gene sequence into a target cell. It does nothing to address the biggest stumbling block of gene therapy. While this is sexycool news, being cured for 3 or 7 months doesn't mean being cured for life.
Claimer: IAAMD
I don't mean to be a downer. We're just a loooong way off from real gene therapy.
Fermat's other theorem: "I have a simple proof, but I can't write it down as I fear it's a DMCA violation to discuss it"
A stem cell is cell that can turn into different type of cells. There are many type of stem cells, and the controversy in the US is only over human embryonic stem cells. These cells can only be obtained by destroying what many consider a human life.
Stem cells in adult bone marrow can turn into many types of blood cells. From the article, it sounds like the stem cells used came from the patients' own bone marrow so human embryos probably weren't used. The article doesn't say where the normal copy of the gene came from, but I doubt it would need to come from a human embryo.
The problem with a retro-virus, is that each time it replicates, it takes a small chunk of the host's dna with it. Each iteration of the virus is a different virus altogether. Hence, the problems with developing a vaccine or cure. Influenza and HIV are both retroviruses. Google knows. Look up 'retro-virus/immunology' if you're interested in more reading...
-1 offtopic.
Retroviruses are being investigated for 3 reasons:
1) They can be used as vectors to transport genetic information into a host cell.
2) Reverse transcriptase can be used to isolate DNA sequences from a mRNA chain so that the gene can be manipulated through bioengineering techniques.
3) To find a way to genetically engineer a cure for AIDS. If the action of reverse transcriptase can be halted somehow, the HIV virus will have no way to spread its harm through the body and millions of lives could be saved.
more info
Retroviruses are cute little viruses that write DNA from RNA using reverse transcriptase, an enzyme. These viruses possess the ability to write that DNA into pre-existing DNA and, in this manner, convert cells and such to their cause. HIV is a retrovirus. However, much more beneficial retroviruses exist. The ability to write DNA into cells allows these viruses to be used to modify live cells. Take this with a grain of salt: I've never been a very good Bio student.
Pax Digitalia
I don't remembering God *having* a biotech lab, or at least it wasn't mentioned in any bible *I'VE* ever heard of... (Though, perhaps it's in $cientology's secret documents)
UNTIL we can manipulate ALL REALITY with only the power of WILL, we will NOT be be coming anywhere close to "playing god".
"Your superior intellect is no match for our puny weapons!"
And how is genetic engineering (or, at least, the type described in the article) disturbing? It almost certainly saved this baby's life, and prevented him from suffering a short, isolated existence in a plastic bubble, not to mention the psychological trauma of an accidental viewing of this piece of dreck.
This gives a great example of the safer of the two types of gene engineering, somatic. This type of gene therapy only modifies the genetic makeup of certain cells in the body. None of the effects of the changes could propogate onto his children. I wish we could see more of this type of gene therapy.
The other type, germline, alters genes in gametes (eggs and sperm). Any changes here would probably (at least with our technology) be irrevsible and would be carried by any decendents. Thankfully, people are being more cautious with this kind since the effects would be much more permanent and far reaching.
Nope, you're pretty much right about a retrovirus. My Bio teacher came up with a really great analogy about retroviruses, HIV in particular: Ok, say you've got eight men in a tank. These guys each have a set of blueprints. They drive their Panzer tank through the wall of the nearest Ford factory and tell the workers to make more tanks instead of those fruity Ford Taurus things. The workers construct 7 more tanks and the guys each hop into their respective tanks, driving out of the building through the walls, bringing it to the ground. You've got the tanks as the delivery system, the men as the viral RNA, and the factory as the cell. Mind you, I may have skewed it slightly because it's been a good three months since we covered that. Oh well, it's also tired time. Heh.
If I wasn't so lazy, I'd have a sig.
While this was going on there is a couple in California that is hopeing (as in activly looked for sperm donner who was deaf) to have a kid that is deaf so that he will be like the rest of the family (minus the cats). So while we have gene experaments going on to inhance the lives of people and potentialy bring a brave new world kind of classism effect [BadThing(TM)] we have also got people who are actively trying to set the pace of progress back.
Ascii artist &
Remember that one of the arguments against vaccination when it was discovered, was that we shouldn't be "playing god". Eg people should just accept death by lethal contagious viruses like smallpox -- vaccination is "playing god".
Just about every significant medical discovery has been opposed with the "playing god" argument.
Should have to go to talk to the person cured, and explain why they think not doing this research is more important to their values than him being cured.
dominionrd.blogspot.com - Restaurants on
But we'll also develop targeted bioweapons to kill "terrorists","Dangerous Radicals", Saddam Hussein, or other enemy-of-the-year. We'll do horrible shit with this, possibly doomsday our species along with our environment.
I got the two states interposed..
A Zygote (fertilised ovum, basically) *precedes* a blastocyst, which, in turn, precedes an embryo.
Sorry 8P
Brak: What's THAT?
Thundercleese: A light switch.. of TOTAL DEVASTATION!
In all cells things go like this:
DNA -> RNA -> Protien
aka the central dogma of biology
A virus is incapable of doing this by itself, hence it cant reproduce by itself.
So it hijacks the host cell.
There are several classes of virus, based on
what it injects into the host cell. Some have subclasses that are based mostly on what it looks like (capsid and envelope) of the virus.
I. dsDNA
(papovirus) warts
(adenovirus) respiratory disease
(herpesvirus) herpes, chickenpox
(poxvirus) smallpox, cowpox
II. ssDNA (parvovirus)
roseola
III. dsRNA (reovirus)
diarrhea viruses
IV. ssRNA that can serve as mRNA
(picornavirus) polio, common cold
(togavirus) rubella, yellow fever
V. ssRNA that is a template for mRNA
(rhabdovirus) rabies
(paramyxovirus) measles, mumps
(orthomyxovirus) Influenza viruses
VI. ssRNA that is a template for DNA synthesis
(retrovirus) HIV, tumor viruses
The Retroviruses work something like this:
RNA -> DNA -> RNA -> Protien
This is a case where biology doesnt follow the central dogma of biology! The other virus classes still follow the central dogma.
Another interesting disease agent is a prion, but thats involves a lot of speculation.
-hope that helps.
I should hope not.
People normally have 23 pairs of chromosomes. Of those chromosomes, only 1 is an X chromosome, and one a Y chromosome in boys. Girls have 2 X chromosomes.
The other 22 pairs of chromosomes may be X-shaped, but they most assuredly not what biologists refer to as X chromosomes; they're referred to by pair number 1 through 22.
Furthermore, having an abnormal number of chromosomes (aneuploidy) can cause congenital disease. In the case of sex chromosomes, I refer you to Klinefelter's and Turner's syndrome. In the case of other chromosomes, Down's syndrome (extra copy of chromosome 21), trisomy 13 and 18 (extra copies of chromosome 13 or 18).
Sheesh.
The other 22 pairs of chromosomes may be X-shaped, but they most assuredly not what biologists refer to as X chromosomes
:)
Ok...
Now that you mention it...I vaguely remember one of my college teacher saying something to that effect...
I often have trouble expressing to people why I left science classes and went fort art college instead...I think I'll user this example from now on. Its just cute enough
sheesh...
You can't take the sky from me...
X chromosomes are distinctly different from the autosomal chromosomes. No human being can live with a missing autosomal chromosome (e.g. only one chromosome 21 instead of two) -- embryos with this type of defect are miscarried so early that they are not even detected, even though embryos with three copies of an autosomal chromosome (a defect arising from the same mistake in meiosis which causes the loss of an autosomal chromosome in some embryos) are detected -- some even live to adulthood (Down syndrome). On the other hand, all human beings can be said (in general) to have only one X chromosome; in females, one X chromosome is almost completely inactivated in each cell.
Name me one thing in nature we fully understand. Name me one thing.
We don't know, *for sure* how atoms work or are built. We don't know if there is a 5th repulsive force in nature. There's lots we don't know..
But what we do know.... To our knowledge, this therapy may help a guy who's *never* had a chance to go out into real life. Maybe it'll give him cancer in 30 years. Maybe it won't.. But just because it might possibly be catastrophic doesn't mean that nothing should be done.
That way leads to stagnation and helplessness. We don't know and can't know. That is why this so-called 'precautionary principal', that something must be proved 'safe' before it can be used or sold is garbage. We can't know and won't know for *sure* anything.
Gene therapy
Similar method was originally tried on cystic fibrosis patients, but the positive results lasted only for about three weeks, after that repaired cells were replaced back again with the faulty ones.
It seams to be more complicated.
Cystic Fibrosis Gene Therapy
Read the article. Embryonic stem cells were not used. They used his own stem cells.
News for Nerds. Stuff that Matters? Like hell.
Great English explanation :)
Does anyone think it is a good start, towards the cure of all genetic diseases passed to boys just because we don't have a backup copy of X?
Looking towards the day when difference in life expectancy between the male and the female is negligible.
You would have to use a recombinant vector, i.e. a vector which has the start and end of the gene for the correct receptor with it, and some deletion or garbage in between. It _could_ recombine, but would not work for a multi-cellular organism, since only part of the cells would become immune.
The idea of dual complex treatment is just a bit to much at this moment if you'd ask me.
and yes, I'm a Molecular Geneticist
Sig (appended to the end of comments I post, 54 chars)
I'm not suggesting for a minute that we stop. I am truly in awe of what they have accomplished, and the incredible potential for improving human lives. I thought this was an exciting story, and I am happy for the boys who suffer from this disease. Maybe it's because I'm more of a physical sciences kind of guy, but thought of being able to mainipulate individual human genes, effectively retroactively as I understood this, is just mind-boggling. If we're advanced enough to pull this off, are there any limits to what we can do?
And that is where the negative side of my comment comes from. What are the limits to what we can do, and (rhetorically) are we up to the responsibilty? The answer is "no" - though the prospects for good are unlimited, some will abuse this technology. It's the inevitable cloud that accompanies the silver lining.
In my opinion, that's part of the price we pay for advancing. Genetic manipulation seems much like our first steps into atomic power (another subject that provoked fears of "playing God"). It is far more revolutionary than medications or cutting trees or most of the other ways we manipulate our world. These other things can have tremendous cumulative effect due to scale, but their potential individually is fairly narrow and limited. A new drug may heal - or hurt - a few individuals, but it can't change the shape of the human race.
Genetic manipulation is different. It can literally change the face of humanity. The potential for good is awesome, but it will come with a price. And that's the risk we accept every time we move forward.
Again, sorry for provoking a religious discussion. My use of "playing God" was only meant as a metaphor for the power and potential of this development.
Blood tests!
White blood cell count.
And they can also see if the white blood
celss are efficient is a test tube.
http://www.donarmstrong.com
It wouldn't be a cure, but if you created a
new line of stem cells in the bone marrow that
produced HIV resistant T-cells, the HIV infected
person would never lose his/her immune system,
and so live a nearly normal life.
But they would still be capable of passing on
HIV though.
Can this be used to cure Aids as well? I mean, honestly, I've asked myself a couple of times, if I ever got AIDS, why couldnt I have a bubble? Isn't having bubble boy disease similar to AIDS? (no immune system?) I mean, instead of taking drug cocktails, why not live in a bubble?
If you're not a Liberal in your 20's, then you have no heart.If you're still a Liberal in your 30's you have no brain.
Yeah, blastocysts have differentiated tissue types. Zygotes are one cell, I believe.
toeslikefingers.com - because
Well technically, manipulating all reality through the power of will alone is a pretty good functional definition of what it means to BE God.
This is all very simple until things get polytheistic:
So what about the lesser gods? Like the little ones who make the bus come in time and protect rivers etc? I thought you could define god as anything that's not actually human ( or !cowboyneal)).
Also, "creative gods" doesn't mean they also own everything they created. So I'm not proprietary humanware and I'm recursively able to create and play too, thank you very much. Regardless of who created me.
Then again, if it was greek gods, I'm sure you'd get loads of bitching about who created who, and some crap about zeus laying claim to all the other guys and creating proprietary viral licensing etc. Next thing you know that other guy would start firing his lighning bolts and chaos ensues. What a bore. That's what you get from gods who sit around together all the time. Bit like big brother the other way round.
Better to have gods who are a little more isolated but more independant and with a little more space to work in. Maybe there's a god of only science, and scientific exploration, enemy but mostly tolerant of the god who protects spiritual hippy dippy shit. Even Linus and open source are seen as god and religion respectively by some. One god. That's monopoly.
Ale
Let's assume we believe in the existence of a supreme being in the Western Judeo-Christian tradition. What is His nature? To create the world and set the rules by which it functions, and then leave its inhabitants to exercise their free will within it.
And what is the nature of man? To strive to understand the rules by which nature works, and do everything in his power to exploit those rules to his own ends.
In short, it is the very nature of God to allow events to happen without his direct intervention. It is the very nature of man to attempt to control events.
"Playing God" would be letting someone die even though we can save him.
Nope, no sig
As in favor as I am of stem cell and other genetic research and, more importantly, applications being found for the results of that research, curing a disease that causes such a massive immune system failure has to be done at the source: it's a genetic thing, as far as I know, so we have to let these people die. Keeping them around may be humanitarian. Curing their disease may make living worthwhile and hopefully can let them contribute to society. Letting them reproduce, however, will weaken our gene pool; and now that they can come out of their bubbles, they'll be reproducing even more. Just like cancer cells in an otherwise healthy body: the unwanted units become more and more pervasive and harder to contain or remove.
As well as the retro virus taking host DNA (see other reply), there is another factor at work.
Most anitbiotics, and antvirals, work by stopping the infectious agent from reproducing, and it is the hosts immune system that kills the remaining infectious agent. HIV decimates the immune system. This doesn't help.
Michael J. is a perfect example. He's a perfect example IMO, this was a good guy struck down by a disease that many of us know little about. To watch him fight this is truly heart breaking and at the same time inspiring. I don't know your grandmother but I'm sure there are many others out there just like her and if we can find a way to prevent or cure diseases like that I think we should try. No one I know wants to end up invalid in a bed struck down by something like this - I'd rather die quickly. My grandfather is slowly slipping, not a particular disease just old age has taken it's toll. It breaks my heart and that of my family to watch it. This man was a rocket scientist, literally, who helped get the Posiden (sp?) missle program going and who did lots of engineering things during WWII. To watch him lose his mind is awfull.
Those who would cry out against helping people like these kids should try one of hese diseases on for size themselves. I fully understand that such research could also lead to destructive things but not trying to help could be nearly as bad. We simply have to hope that people use common sense and proceed slowly. Unfortunatly I think common sense is in shorter and shorter supply these days (sigh)....
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Someone mod this +1 Inciteful
Amen.
Play Command HQ online
I assume you are refering to Western Europe or the United States in you weapons argument. There are several international bans on biological weapons...
I was referring to the United States military, primarily. I was raised in a military family, and played soldier for a while. If something is weaponable it will be used. In the early 80's I was into RC planes in a big way. We had a squadron of planes - mostly junior NCO GIs and brats - and discussed military applications of the technology. I should have wrapped the whole post in <rant></rant> tags. :)
(the United States has not engaged in offensive microbiological weapons since the Nixon administration)
Uh, right.
Also I must ask what you mean by "targeted" bioweapons. We do not posess the technology to "target" a weapon beyond the scope of what species it effects (which is usually a trait inherited from the natural stock microbe). It is (and will remain) impossible to target a virus or bacterium to kill one man or nationality.
Thanks, I didn't now that. I'm not a microbiologist so I will defer to your expertise. I had assumed that it would eventually be possible to target specific individuals via a DNA fingerprint of some sort.
Don't get me wrong. I'm all for stem cell research. I'm happy and amazed that Rhys Evans aka "Bubble Boy"was cured. I hope it advances medical science by an order of magnitude on the technology scale.
However, I do believe that if it's practical to weaponize something, it will be done. Technology in and of itself is neither good, nor bad. Ethics can only be applied to how it is used.
No doubt, we will do great good with biotech. Hopefully we will avoid any potential catasrophes along the way.
I do believe that the reason Sadam Hussein is the enemy of the year is HIS desire to develop bioweapons.
Yes, that and other things. Saddam Hussein is not a warm and fuzzy teddy bear. He's an asshole, but an effective and powerful dictator. He stood up against the United States and a "coalition army" and remains in power. He's well-respected for that - even if he's not loved.
There were political reasons why we didn't knock him out the last time. We would have pissed off his neighbors who are already massively supsicious and resentful of our military presence where they live.