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Actual Gene Editing in a Patient with Metabolic Disease

Discussion in 'Other Health News and Research' started by anciendaze, Dec 2, 2017.

  1. anciendaze

    anciendaze Senior Member

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    This story turned up in an unusual place, Wall Street Pit, because of interest by venture capital firms. It states that a patient with an incurable inherited metabolic disease, Hunter syndrome, has been successfully treated using CRISPR gene editing. There is obviously far more to know about this report, but I'm starting this topic to have a place for information on this subject.
     
    Countrygirl, *GG* and Gemini like this.
  2. Marky90

    Marky90 Science breeds knowledge, opinion breeds ignorance

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    Well let`s see if hes cured before calling it a breakthrough?:p Genetic repair is the future tho
     
  3. drob31

    drob31 Senior Member

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    I'm curious how it would edit the genes in all 100 trillion cells?
     
  4. anciendaze

    anciendaze Senior Member

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    If you did that, you would likely change germ-line cells, producing a heritable change. Doing so would cross an ethical boundary, since unborn children could be affected by an experiment performed before they were conceived. This is a much more modest change. You also need to remember that there are hundreds of types of cells, and most genes are only active in a limited number of cells of some types. Simply editing a gene in those cells where it is actively transcribed would result in cures for many conditions.
     

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