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CRISPR Therapy Cures First Genetic Disorder Inside the Body

posted by mrpg on Saturday July 31 2021, @05:13AM   Printer-friendly
from the and-the-mind? dept.

upstart writes:

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)

Original Submission

 
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  • (Score: 4, Funny) by Rosco P. Coltrane on Saturday July 31 2021, @08:10AM (3 children)

    by Rosco P. Coltrane (4757) on Saturday July 31 2021, @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.

    Starting Score:    1  point
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  • (Score: 1, Funny) by Anonymous Coward on Saturday July 31 2021, @09:06AM (2 children)

    by Anonymous Coward on Saturday July 31 2021, @09:06AM (#1161657)

    C-x C-s

    • (Score: 5, Funny) by Rosco P. Coltrane on Saturday July 31 2021, @09:16AM (1 child)

      by Rosco P. Coltrane (4757) on Saturday July 31 2021, @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 2021, @04:11PM

        by Anonymous Coward on Saturday July 31 2021, @04:11PM (#1161727)

        Finger Acrobatics is a new Olympic event.