Slashdot Mirror

← Back to Stories (view on slashdot.org)

Gene Therapy Cures "Bubble Boy"

Posted by michael on 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."

6 of 362 comments (clear)

  1. More coverage... by abhinavnath · · Score: 5, Informative

    The Guardian, and Yahoo

    --
    My other sig is also a .Porsche
  2. 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
  3. 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.
  4. 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"
  5. 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

  6. 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.