CRISPR gene editing breakthrough could treat many more diseases – Engadget
CRISPR gene editing already promises to fight diseases that were once thought unassailable, but techniques so far have required injecting the tools directly into affected cells. That's not very practical for some conditions. However, there's just been a breakthrough. NPR reports that researchers have published results showing that you can inject CRISPR-Cas9 into the bloodstream to make edits, opening the door to the use of gene editing for treating many common diseases.
The experimental treatment tackled a rare genetic disease, transthyretin amyloidosis. Scientists injected volunteers with CRISPR-loaded nanoparticles that were absorbed by the patients' livers, editing a gene in the organ to disable production of a harmful protein. Levels of that protein plunged within weeks of the injec...