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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."

13 of 362 comments (clear)

  1. No cure by DeadBugs · · Score: 5, Funny

    Sadly there is no cure for the people who got sick after seeing the movie "Bubble Boy"

    --
    http://www.kubuntu.org/
  2. Perhaps now... by funkbrain · · Score: 5, Funny

    ...science can answer the Moors/Moops riddle which has plauged mankind since the dark ages!

  3. Question by Da+Masta · · Score: 5, Interesting

    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?

    1. Re:Question by glwtta · · Score: 5, Informative

      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.

      --
      sic transit gloria mundi
    2. Re:Question by ageitgey · · Score: 5, Informative

      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 :)

      --
      Uninnovate - Only the finest in engineering.
    3. Re:Question by nathanm · · Score: 5, Informative
      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.
  4. More coverage... by abhinavnath · · Score: 5, Informative
    --
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  5. gene therapy by borg · · Score: 5, Informative

    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"
  6. Re:Playing God? by flynt · · Score: 5, Insightful

    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.

  7. retrovirus information by jest3r · · Score: 5, Informative
    A retrovirus is special because it contains an enzyme called reverse transcriptase. This enzyme works backwards, translating RNA 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

  8. Re:Playing God? by bani · · Score: 5, Insightful

    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.

  9. Name me one... by Convergence · · Score: 5, Insightful

    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.

    1. Re:Name me one... by k98sven · · Score: 5, Insightful

      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.