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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: -1, Troll) by Anonymous Coward on Sunday August 01 2021, @07:09PM

    by Anonymous Coward on Sunday August 01 2021, @07:09PM (#1162018)

    God, I HATE internet experts.

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