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posted by mrpg on Saturday July 31, @05:13AM   Printer-friendly [Skip to comment(s)]
from the and-the-mind? dept.

CRISPR therapy cures first genetic disorder inside the body:

A new CRISPR therapy: Now, researchers from Intellia Therapeutics and Regeneron Pharmaceuticals have demonstrated for the first time that a CRISPR therapy delivered into the bloodstream can travel to desired tissues to make edits.

"This is a major milestone for patients," Jennifer Doudna, co-developer of CRISPR, who wasn't involved in the trial, told NPR.

"While these are early data, they show us that we can overcome one of the biggest challenges with applying CRISPR clinically so far, which is being able to deliver it systemically and get it to the right place," she continued.

Journal Reference:
Julian D. Gillmore, Ed Gane, Jorg Taubel, et al. CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis, New England Journal of Medicine (DOI: 10.1056/NEJMoa2107454)


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  • (Score: 4, Funny) by Rosco P. Coltrane on Saturday July 31, @08:10AM (3 children)

    by Rosco P. Coltrane (4757) on Saturday July 31, @08:10AM (#1161654)

    CRISPR therapy delivered into the bloodstream can travel to desired tissues to make edits

    Don't forget :wq when it's done, else it's all for nothing.

    • (Score: 1, Funny) by Anonymous Coward on Saturday July 31, @09:06AM (2 children)

      by Anonymous Coward on Saturday July 31, @09:06AM (#1161657)

      C-x C-s

      • (Score: 5, Funny) by Rosco P. Coltrane on Saturday July 31, @09:16AM (1 child)

        by Rosco P. Coltrane (4757) on Saturday July 31, @09:16AM (#1161658)

        No, sorry. We're talking about serious, real engineering here, not finger acrobatics.

        • (Score: 0) by Anonymous Coward on Saturday July 31, @04:11PM

          by Anonymous Coward on Saturday July 31, @04:11PM (#1161727)

          Finger Acrobatics is a new Olympic event.

  • (Score: 3, Interesting) by HiThere on Saturday July 31, @01:22PM (3 children)

    by HiThere (866) on Saturday July 31, @01:22PM (#1161684) Journal

    The liver is where just about anything put into the bloodsteam ends up. And most of it gets degraded there. I can see this approach working to treat things affecting the blood or the liver, and just about nothing else with current technologies. (Skin could be handled with a cream. Another exception is direct injection into solid tumors.)

    --
    Javascript is what you use to allow unknown third parties to run software you have no idea about on your computer.
    • (Score: 5, Interesting) by Samantha Wright on Saturday July 31, @05:32PM (1 child)

      by Samantha Wright (4062) on Saturday July 31, @05:32PM (#1161747)

      A cream probably wouldn't penetrate deeply enough to have staying power on the adult stem cells for skin. A better target for surface application would be in the intestinal lining, e.g. for treating coeliac disease. CRISPR has also successfully been used with bone marrow transplants to alleviate sickle-cell anaemia. More generally, consider the various kinds of therapies that exist which already successfully isolate various organs and tissues: for example, patients with cystic fibrosis could have their lungs removed, bathed in a suitable solution, and re-implanted, quite similarly to how they are currently treated with donor lungs. Any transplantable organ can likely be treated with the methods showcased in this paper.

      • (Score: 2) by HiThere on Saturday July 31, @11:17PM

        by HiThere (866) on Saturday July 31, @11:17PM (#1161802) Journal

        When you're considering "removing the lungs" as a part of the treatment I suspect you're going well beyond anything that's widely applicable. The point about the intestine was valid, though, And I was considering the use of "penetrating agents" with the cream, but the point was that the treated area is directly addressed, not via the blood stream. Getting a treatment through the blood stream and past the liver is a real problem.

        --
        Javascript is what you use to allow unknown third parties to run software you have no idea about on your computer.
    • (Score: -1, Troll) by Anonymous Coward on Sunday August 01, @07:09PM

      by Anonymous Coward on Sunday August 01, @07:09PM (#1162018)

      God, I HATE internet experts.

  • (Score: -1, Spam) by Anonymous Coward on Saturday July 31, @05:27PM

    by Anonymous Coward on Saturday July 31, @05:27PM (#1161744)

    Now just target melanin-producing genes, mix it in with a mandatory vaccine, and you've solved America's negro problem.

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