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posted by mrpg on Saturday July 31 2021, @05:13AM   Printer-friendly
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: 5, Interesting) by Samantha Wright on Saturday July 31 2021, @05:32PM (1 child)

    by Samantha Wright (4062) on Saturday July 31 2021, @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.

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  • (Score: 2) by HiThere on Saturday July 31 2021, @11:17PM

    by HiThere (866) Subscriber Badge on Saturday July 31 2021, @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.

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