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Science

Gene Therapy Cures "Bubble Boy" 369

Posted by michael from the next-we'll-see-if-hemos-can-be-cured dept.
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."
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Gene Therapy Cures "Bubble Boy" More

Gene Therapy Cures "Bubble Boy"

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  • Guess I was wrong! (Score:3, Funny)

    by tcd004 ( 134130 ) on Wednesday April 03, 2002 @11:11PM (#3282040) Homepage
    I thought he was cured by scientology!

    VISA's cure for celebrity shoplifters... [lostbrain.com]

    tcd004
  • Now can they just make me seven feet tall so I can play pro basketball?

  • Great! (Score:1)

    by h4l0 ( 561266 )
    Im really happy that they were able to cure "bubble boy". hopefully this means that other oppritunities to cure people with "Gene Therapy"

  • No cure (Score:5, Funny)

    by DeadBugs ( 546475 ) on Wednesday April 03, 2002 @11:14PM (#3282053) Homepage
    Sadly there is no cure for the people who got sick after seeing the movie "Bubble Boy"

  • Perhaps now... (Score:5, Funny)

    by funkbrain ( 217835 ) <nathan_g.fastmail@fm> on Wednesday April 03, 2002 @11:14PM (#3282056)
    ...science can answer the Moors/Moops riddle which has plauged mankind since the dark ages!

  • Question (Score:5, Interesting)

    by Da Masta ( 238687 ) <dmu_netNO@SPAMhotmail.com> on Wednesday April 03, 2002 @11:15PM (#3282058)
    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?

    • Re:Question (Score:5, Informative)

      by glwtta ( 532858 ) on Wednesday April 03, 2002 @11:19PM (#3282073) Homepage
      The laws don't restrict the research itself, they restict the number of cell lines available for research. So yeah, it would've been possible in the US, provided the scientists could get their hands on the stem cells.

      • Re:Question (Score:5, Informative)

        by nathanm ( 12287 ) <nathanm.engineer@com> on Wednesday April 03, 2002 @11:47PM (#3282198)
        The laws don't restrict the research itself, they restict the number of cell lines available for research.
        They don't actually restrict the number of stem cell lines, they merely limit government funding to the existing lines. Anyone could start new lines with private funding.

        Also, the policy only refers to embryonic stem cells. The bubble boys were cured with their own stem cells.
        • That is true, however the effect is still that the overall number of (embryonic) cell lines is smaller, which was my point ;)

        • Re:Question (Score:2, Informative)

          by WowTIP ( 112922 )
          Anyone could start new lines with private funding.

          Afaik that is not true. Bush banned the development of new embryonic stem cells ~6 months ago. The congress is even discussing a ban on importing embryonic stem cells.

          Sure, they could always try to get new lines from adult stem cells, but those attemps has so far not been very successful.
          • Bush banned the development of new embryonic stem cells ~6 months ago.
            No, all he did was block them from federal government funding. You could still use private funds, or even state or local government funds to start a new line. However, state & local governments don't spend much, if anything, on scientific research.

            There are plenty of billionaires that could fund the research though, but none that are serious about fighting disease. Bill Gates only donates money when Microsoft is getting bad press. If Ted Turner, Warren Buffet, or he were diagnosed with some terminal disease, all of a sudden there would be more funds than scientists knew what to do with.
        • "The bubble boys were cured with their own stem cells."

          It looks like you are only half-right. They added complete genes for immune system production to the stem cells. Where did they get those genes?

          I really want to know where they got the retro virus too. :)

    • Re:Question (Score:5, Informative)

      by ageitgey ( 216346 ) on Wednesday April 03, 2002 @11:27PM (#3282109) Homepage
      The US has laws limiting embryonic stem cell research. They don't care if use use cells from yourself (as they did with the 'bubble boy'). The issue is whether or not more break-throughs of this type could be made faster by using stem cells from all the frozen embroyos laying around (which are basically big clumps of stem cell :)

    • Yes, Maybe (Score:2, Informative)

      by Shook ( 75517 )
      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.
    • I think a more important question is, have the Executive orders restricting research and funding caused enough good researchers working on stem cells, embryonic or not, to leave the US and go to the UK, or parts of the EU, where there are less restrictions? Thereby causing the US to fall behind in the tech needed to be able to do this?

      Although the biggest complaint is from embryonic stem cells, Congress is going to be discussing that and theraputic cloning soon. If they ban that it is only a hop skip and a jump away from banning research like this which uses genetic modification. I know a few biologist who are worried about this and are thinking of leaving the country. Is our government causing a brain drain?

  • More coverage... (Score:5, Informative)

    by abhinavnath ( 157483 ) on Wednesday April 03, 2002 @11:15PM (#3282060)
    The Guardian [guardian.co.uk], and Yahoo [yahoo.com]

  • In a related story... (Score:3, Funny)

    by cosmicg ( 313545 ) on Wednesday April 03, 2002 @11:16PM (#3282063)
    Paul Simon has announced a full recall of his "Graceland" CD...

  • Playing God? (Score:2, Insightful)

    by Bowfinger ( 559430 )
    Part of me is terrified at the potential for creating unimaginable horrors; the other part is completely in awe of the amazing things science can do. We're moving closer and closer to playing God. I pray we're up to the responsibility.

    Truly awesome.

    • Re:Playing God? (Score:5, Insightful)

      by flynt ( 248848 ) on Wednesday April 03, 2002 @11:36PM (#3282147)
      Why is it that no matter what things humans do to the earth (good or bad), the ONLY time we're "playing God" is when we fiddle with genes. Very arbitrary criterion if you ask me. Did not God create the trees and the animals? Why when we destroy or create these things then are we not "playing God". It seems a bit illogical to me.

      • Re:Playing God? (Score:5, Insightful)

        by bani ( 467531 ) on Thursday April 04, 2002 @12:30AM (#3282403)
        When vaccination was discovered, the religious community spoke out against vaccination on the same grounds -- that dying of smallpox was "god's will" and that vaccination was "playing god".

        The claim is as stupid today as it was then.
        • When vaccination was discovered, the religious community spoke out against vaccination on the same grounds...

          A nitpick, but an important one: saying "the religious community" is like saying "the Slashdot community."

          My religion did nothing of the sort that you describe. I'm sure the majority of the rest of them didn't either. Unfortunately, as with any perceived demographic, it's the most extreme kind that appoint themselves to speak and act for the rest of us.

      • How about "playing god", not "Playing God" (Score:4, Insightful)

        by Bowfinger ( 559430 ) on Thursday April 04, 2002 @04:19AM (#3283061)
        FWIW, my comment was taken more literally and more negatively than I intended. Sorry for provoking people.

        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.

      • Did not God create the trees and the animals?

        Nope. Unless you care to prove it?

      • Re:Playing God? (Score:4, Insightful)

        by Chanc_Gorkon ( 94133 ) <gorkon@gmaiHORSEl.com minus herbivore> on Thursday April 04, 2002 @07:55AM (#3283543)
        God gives us everything. At least that's what I belive. If god does not want us to know something, he won't let us. That's my opinion. So long as any gene research does not result in a death or crippling some one or for the sake of better boobs, I think it's ok in my opinion. I personally would have loved some of this stuff to be found out years ago because now I may have a grandma to talk to and her talk back instead of visiting someone who can just listen to me. I am not sure what she had, but what I do know is it messed her brain up and she's almost not like grandma. All of her mental functions are ok, it's just that she doesn't have much control of her muscles. It started as a flayling of hands....for a time they thought it might have been Parkinsons, but it wasn't. Now you have to be careful how close you get to her sometimes cuz she could accidently slap ya. Not funny and she doesn't mean to do it, but if they had figured out how to fix this in time, she'd be mostly normal (even if she's old). I want to WALK with my grandma and have fun with her like I did when I was a kid/teenager. My son will only know his great grandma as some old lady in a bed. If any religious zealot wants to keep this from people who truely need it, then to heck with em! How many years more of Michael J Fox's acting would we have if they could reverse his parkinsons? I think it's right as long as we are fixing something. Doing genetic alterations in the womb is something that should not be done and also gene alterations just for better looking kids is a bad thing too. Genetic alteration to fix something that could hurt someone all of their life, well, I feel a bit different about that cuz if this stuff would have happened sooner, I would probably still have a vibrant grandma instead of a invalid grandma.
        • 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)....

      • 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.
    • Are we really playing god in any new way? Sure there is a lot of power in genetic treatments, but transplants, antibiotics, and other major medical advances were also amazing leaps at the time. Its taking so long to develop these new treatments that I think we'll get it right.

    • God's Biotech Lab... (Score:3, Insightful)

      by gnovos ( 447128 )
      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".

      • Re:God's Biotech Lab... (Score:4, Interesting)

        by dragons_flight ( 515217 ) on Thursday April 04, 2002 @01:28AM (#3282658) Homepage
        Well technically, manipulating all reality through the power of will alone is a pretty good functional definition of what it means to BE God. Only important thing missing is knowing everything, but that probably comes along with all events playing out according to one's will.

        As you no doubt know, "playing god" is what the disdainful call it every time man gains control over an aspect of reality that was previously ruled by chance/God alone.

        Since God created us with intellect, reason and the ability to learn moral judgment, I for one, believe that we should "play God". Man is created in the image of God, and if we are to fulfill that destiny than it means learning to act with as much wisdom, knowledge and moral judgment as humanly possible, which certainly includes scientific exploration.
        • 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

        • Well technically, manipulating all reality through the power of will alone is a pretty good functional definition of what it means to BE God. Only important thing missing is knowing everything, but that probably comes along with all events playing out according to one's will.

          Sure - if you can manipulate reality by power of will alone, it should be dead simple to will yourself all the knowledge of the universe =)
    • Part of me is terrified at the potential for creating unimaginable horrors; the other part is completely in awe of the amazing things science can do. We're moving closer and closer to playing God. I pray we're up to the responsibility.

      If humans can do it, then it's hardly inspiring enough to be "playing God".

    • Uh, no. (Score:4, Insightful)

      by bani ( 467531 ) on Thursday April 04, 2002 @12:28AM (#3282388)
      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.
      • On March 8, Peggy Noonan had a particularly wonderful article in the Opinion Journal. Within that article [opinionjournal.com], she had these things to say:
        I am experiencing a change of temperament, if that is the word. I have mostly gone through life as a short-term pessimist and a long-term optimist, but now I find, and perhaps it's only temporary, that I am increasingly a short-term optimist and a long-term pessimist. That's not quite right. I am certain there is a heaven, which is not a pessimistic belief. But my long-term thoughts about the world are not as sunny as once they were.

        And yet I am happy each day and enjoy my life.

        While I am worried about the future in a way I cannot shake.

        ...

        The friend who had e'd me followed up with news that the Chinese are creating dozens of cloned embryos in their labs. The British medical journal New Scientist has reported a Chinese team "based at Shanghai No. 2 Medical University" says it has "derived stem cells from hybrid embryos composed of human cells and rabbit eggs." The journal said scientists throughout the world fear similar research in the US and UK has been "bogged down" by "ethical concerns."

        Ah, those pesky ethical concerns. They slow you up just when you could be creating in a Petri dish the recipe for Rabbit Man. And then of course you could grow him, bring him into being, for all but dunces know that what man can do he will do(*). And then perhaps once you've grown him you can have Rabbit Man for dinner.

        (* - My emphasis, not original)

        I certainly don't think that this is the step that is going to break the camel's back. Clearly, this is an awesome and great and GOOD achievement. And we will do great things in this fashion. But research like this requires great responsibility, and not everyone will understand that.

    • Being God (Score:2, Interesting)

      by Sean Clifford ( 322444 )
      We're not going to use this technology responsibly - we don't use any technology responsibly. Of course, we'll develop "good" uses for the technology - treat and cure diseases, discover new forensic techniques, grow new organs, and blah blah blah.

      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.

    • In my opinion, having a death penalty is closer to playing God than gene therapy, since you accomplish a transition (living-dead) that only GOd was traditionally allowed to make, while genetherapy does not change your state (when you're alive, you're alive, when you're dead, you're dead).

  • Good News... (Score:3, Insightful)

    by mrgaribaldi ( 162490 ) on Wednesday April 03, 2002 @11:18PM (#3282071)
    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.
    • Maybe that should say that you fear the use of technology that you don't understand.

      The risks and complexities of delivering genes that every non-"Bubble Boy" person has anyway isn't high on the list of scientific achievements. That they did so only to marrow precursor cells is sensible since they are the only ones that need express the genes and it's easier to target specfic cell groups than the entire body. The surprising part would have been if all the technology worked as advertised and he wasn't cured.

      There are reasons to worry about genetic manipulation, but there is no reason to cry about a new plague in a situation that is well understood, and which any reasonable geneticist would tell you is very low risk. GM foods are far higher risk, and none of those have yet had an effect even approaching that caused by the transplantation of species into environments where they have no competitors.

    • Name me one... (Score:5, Insightful)

      by Convergence ( 64135 ) on Thursday April 04, 2002 @01:33AM (#3282670) Homepage Journal
      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.

      • Re:Name me one... (Score:5, Insightful)

        by k98sven ( 324383 ) on Thursday April 04, 2002 @10:27AM (#3284214) Journal
        We can't know and won't know for *sure* anything.

        How interesting.. this is called relativism (not the Einstein kind)

        Now, I would like to ask you, does that statement apply to itself?
        If yes, then we can't be sure that everything is unsure
        - which renders the possibility that things indeed can be known for sure.

        If no, then you are assuming that at least one thing -can- be known for sure,
        which means that other things may be as well.

        In short: That is a self-contradictory statement.

        Also, in stating that we don't know most things
        -for sure- , you seem to imply that everything is equally uncertain. This is not the case.

        For example, for the last 500 years or so, we have known that the earth orbits the sun, and not vice-versa.
        Of course we can't be -absolutly certain- this is the case, but I'd say that it would be very unlikely for the opposite to be proved.

        Science is not about solid truths, nor has it ever been:
        It's about knowing things with a known degree of certainty.
      • 5th repulsive force

        1. Mariah Carey
        2. Celine Dion
        3. Rosie O'Donnell
        4. Lawyers
        5. Chelsea Clinton
    • Don't apologize, I share your fear regarding potential side effects of such treatments and experiments. I'm all for medical (and scientific) advances, I just hope that in doing so we aren't in fact dooming ourselves to some mass death. (A very good example of something being changed such that it was 'too good' is the recent revelations regarding asthma-- I saw it on CNN I believe, but doctors and scientists believe that some cases of asthma may in fact be due to young children growing up in much more sterile environments. Because the lungs and other internal organs related to breathing aren't exposed to the same level or variety of contaminants, they can't cope with such things once the children start spending more time out in the natural air (it doesn't help that kids also exert a lot of energy, and tend to breathe more during physical activities outside).). That example wasn't related to any one scientific advancement, but it does show that not all things science teaches us is always correct.
  • 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!

  • my experience (Score:2, Informative)

    by azosx ( 568180 )
    I wrote a paper this last semester in college on embryonic stem cell research. The possibilities in this field of research are endless. It's certainly not suprising to me this discovory occured in Europe. It's unfortunate the position the United States has taken towards stem cell research. It's pretty much closed to door and made the possibility of discoveries such as this very unlikely here in the U.S.

    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.

    • Re:my experience (Score:2, Interesting)

      by Anonymous Coward
      They took this guy's stem cells from his own body. Not from some embryo. There are no laws against this in the U.S. What they did was the almost the same thing as how people are treated for Hodgkins disease every day. In simplified form: 1) Extract bone marrow (and thus, stem cells) from patient, 2) Submit patient to massive radiation to kill existing stem cells in his body, 3) Put patient on IV drip of his own bone marrow (Yes, the stem cells "magically" end up where they are supposed to be in the marrow).

      The only difference here (AND IT'S A REALLY COOL DIFFERENCE) is that before they put the stem cells back in this guy, they used genetic engineering techniques to insert a good copy of the Interleukin-2 gene (a "bad" il-2 gene was causing his disease to begin with) into the DNA of the stem cells.

      The majority of the genetic engineering (e.g. recombinant DNA) techniques that made this possible were developed in the US over 20 years ago (and funded by US tax dollars). (These guys appeared to have done some cool things to make the stem cells more likely to be "infected" by the vector.) So don't use this story to make a case against the US policys on embryonic stem cell research. This work has nothing to do with embryonic stem cell research.

      The reason why this stuff probably didn't happen in the US is that our FDA officials are a bunch of overprotective suits. That said, there have been many uses of genetic engineering in the US (treatment of cystic fibrosis comes to mind) to date- although not all have been successful.

      Personally, I think it's awesome that these former bubble boys will have the chance to lead "normal" lives. Everyone involved in getting this done, from the patients themselves to their families to the doctors to the researchers to the governments to whoever paid for it (this must have cost millions including everyting), deserves much congratulations for their vision, courage and hard work.
    • It's certainly not suprising to me this discovory occured in Europe. It's unfortunate the position the United States has taken towards stem cell research. It's pretty much closed to door and made the possibility of discoveries such as this very unlikely here in the U.S.
      The new stem cell policy wouldn't have affected this research at all since it only applies to embryonic stem cells. The bubble boys were cured with their own stem cells.

      Besides, the policy only limits government funding. Anyone could start new stem cell lines with private funds.

  • gene therapy (Score:5, Informative)

    by borg ( 95568 ) on Wednesday April 03, 2002 @11:32PM (#3282132)
    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.

    • Other issues (Score:4, Informative)

      by nucal ( 561664 ) on Thursday April 04, 2002 @05:20AM (#3283218)
      A few reasons why this worked so well:
      • This form of SCID is due to a total gene deletion - so that gene replacement was feasible. A lot of genetic diseases are due to genes expressing proteins that are mutated, but still produced. These mutant proteins can frequently have a dominant negative effect, that is, they mess up normal copies of the protein or other proteins in the cell. In this case, adding more good copies of the gene in question will not help.
      • Being able to treat stem cells in isolation is a big advantage, since you don't have to target cells in the context of the whole body (needle in haystack problem). In culture, the virus to cell ratio can be really high, increasing the probability of successful infection. Also, stem cells successfully expressing the gene of interest can be selected and preferentially propagated in culture before re-introducing into the patient.
      • Another big advantage in being able to treat isolated cells is that you avoid the potential problem of an immune response to the virus itself. This was a big problem [wired.com] recently with a different class of viruses. In some ways SCID, being an immune deficiency, is the ideal disease for viral gene therapy, since these patients are less likely to react to the virus itself.

  • retrovirus information (Score:5, Informative)

    by jest3r ( 458429 ) on Wednesday April 03, 2002 @11:45PM (#3282189)
    A retrovirus is special because it contains an enzyme called reverse transcriptase. This enzyme works backwards, translating RNA [accessexcellence.org] into DNA. Retroviruses contain RNA within their protein coat, and use reverse transcriptase to create DNA that can be inserted into the cell it is attacking. One of the most famous (or perhaps infamous) retroviruses is the HIV retrovirus, which causes AIDS.

    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 [thinkquest.org]

  • 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.

  • Other points (Score:4, Interesting)

    by Faux_Pseudo ( 141152 ) <Faux.Pseudo@g[ ]l.com ['mai' in gap]> on Thursday April 04, 2002 @12:07AM (#3282275)
    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.
    • Washington Post article [washingtonpost.com]. I think they're from the east coast, not CA.

    • Re:Other points (Score:3, Insightful)

      by mekkab ( 133181 )
      Ahhh, I see you read a little washington post magazine while on the can this Sunday!

      However your argument is a gross generalization on both fronts. To go O.T. for sec, the Deaf article is not just commenting on the desireability of a physical conidition (not being able to hear) but of the fierce culture that has associated itself around it. They (the capital-d Deaf) stick together like birds of a feather.
      I don't know how fostering one's own community is stetting the pace of progress back, but YMMV.

      back on topic- to extrapolate from this gene experiment where we have no long term data to establish its true efficacy to a Gattica-type dystopia is almost trolling as a luddite. Just like the "we shouldn't play God!" troll, we have to realize its out of our hands. Technology is neither good nor bad.

      And if you live in America, you have nothing to worry about, since it will either be outlawed, the funding will be cut, or Hollywood will legislate what genes you can use. Whoops, sorry! This isn't a DMCA/RIAA/CBDBTA/TINSTAAFL article!

      • Re:Other points (Score:2, Insightful)

        by ostiguy ( 63618 )
        Dude, when I was in high school, the dumb kids hung out together. Do you advocate allowing parents to genetically engineer dumb kids so the dumb community doesn't get upset?

        ostiguy
        • actually, it's not a personal thing. Personally, I think/feel that the Deaf take it *WAY* TOO FAR to the exclusion of everyone who is hearing. I've heard stories of Deaf parents considering aborting children when they found that their child would come out normal. And comparing dumb kids in high school to a tight knit intergenerational community is pretty ignorant. ostiguy, were you one of those dumb kids? ;)
          (just kidding!)

          And how in hell would those Dumb people figure out how to genetically engineer more Dumb? (that's capital D dumb to you, Mr. Smarty pants!)
          I mean I've seen a fish ride a bicycle but this is ridiculous!

    • I'm not sure how this is crazier than people who assert in polls that, if the technology were available, they would choose to raise only children that were blonde. But then, I consider that pretty dashed crazy.

      (no blonde jokes intended)

  • 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.
  • 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?

  • Survival of the Weakest (Score:2, Insightful)

    by ari_j ( 90255 )
    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.

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