21 votes

First patients to get CRISPR gene-editing treatment continue to thrive

2 comments

  1. Nivlak
    Link
    I myself have a genetic disorder and I am extremely excited to see this kind of thing. The potential of this technology could help so many people, it’s unreal.

    I myself have a genetic disorder and I am extremely excited to see this kind of thing. The potential of this technology could help so many people, it’s unreal.

    5 votes
  2. eladnarra
    Link
    Really cool stuff! I hope more folks with sickle cell disease and similar conditions get access to this relatively soon. I have a mutation that makes me much more likely to get certain types of...

    Really cool stuff! I hope more folks with sickle cell disease and similar conditions get access to this relatively soon.

    I have a mutation that makes me much more likely to get certain types of cancer (and a family history to prove it), so gene therapy is personally interesting to me. I do want to highlight, though, that this particular method doesn't appear to be applicable to many genetic diseases. At least as far as I can tell, based on my somewhat limited background.

    I may be wrong, but it looks like they're doing a technique that lends itself to diseases stemming from blood cells - it's akin to a bone marrow transplant for leukemia, in a way, in that they use chemotherapy to kill off the diseased cells and then replace them with new ones. Only in this case the new cells are the person's own cells which were genetically modified (outside their body, in a lab), rather than a bone marrow donation from another person. There's a relatively long history doing this type of gene therapy (albeit not with CRISPR) for primary immunodeficiencies; the downside was the potential for cancer due to the genetic material being inserted in places that turned on genes that lead to cancer.

    I'm not entirely sure how applicable these findings are for someone like me, for example, who would need to have a gene modified in all (or most of) the cells in my breasts, ovaries, etc to reduce my risk of cancer.

    3 votes