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Gene Therapy Treatment Wins FDA Approval

Discussion in 'Real Life Discussion' started by Xiph0, Sep 1, 2017.

  1. Xiph0

    Xiph0 Administrator Admin

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    Pretty landmark news. This is the crux:

    I have to say I'm surprised by how quick this got approved, since in the past when gene therapies have failed it has tended to be lethal. There's also ethical concerns about directly modifying someones genes in general, especially if the changes you make will be heritable.

    Anyway, this is my field of study. If anyone has questions, AMA.
     
  2. Darth_Revan

    Darth_Revan Secret Squirrel Prestige DLP Supporter

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    Is this CRISPR-based technology?
     
  3. KHAAAAAAAN!!

    KHAAAAAAAN!! Troll in the Dungeon Prestige DLP Supporter

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    Targeted cell infusion gene therapy is a totally different animal than traditional gene therapy (i.e. insertion of new sequences directly into host tissue via viral loads or what have you).

    By only modifying a small cell culture, and then reintroducing those cells into the host (wherein they produce pro-apoptotic effectors or directly kill targeted tissues in the case of these mutated t-cells), you're essentially mitigating the risk of random insertion over wide areas, and only really have to worry about mutagenesis in the cells you injected and/or any daughter lines that arise from those cells.

    TL;DR it is a fairly safe form of gene therapy. Also, would not be heritable unless the t-cells somehow modified gametes (unlikely) or residual modified t-cells were absorbed through placental transfer.

    Edit: I also imagine you would follow up this treatment with a round of immunosuppression to kill off the modified t-cells to reduce risk of lymphoma.
     
    Last edited: Sep 1, 2017
  4. Xiph0

    Xiph0 Administrator Admin

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    Seems likely but the details are probably going to be proprietary information.

    The concern I had in mind wasn't random insertion but immune response if the mutated t-cells are rejected. I expected the FDA to go slowly into the water on this but I think the fact that the labs in China did gene therapy with viable embryos started an arms race:

    Source
     
  5. Download

    Download Chief Warlock DLP Supporter

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    Great to see this happening. Hopefully we can start seeing the worst of genetic diseases wiped out soon.
     
  6. Oz

    Oz Heir to Hogwarts Moderator DLP Supporter

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    Hopefully ginger hair is the first thing to go.
     
  7. Genghiz Khan

    Genghiz Khan Auror

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    As long as we don't go far enough down this line that Huxley starts feeling more prediction and less fiction.
     
  8. Download

    Download Chief Warlock DLP Supporter

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    Yes, resouling those demons is a good first step.
     
  9. Morphe

    Morphe Squib

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    Well I don't really think it's much of a surprise that gene therapy got approved. I mean at this stage gene therapy is the way to go. Right now in med schools gene treatments are feasible study topics in classes and the things you can do are insane if it ever works as predicted. Which it easily might not, as we all know, anything that can go wrong will go wrong.
    To the future, cheers.