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😳 Scientists have, for the first time, applied in vivo DNA base-editing technology directly inside the human body, - Nature
In a clinical trial, researchers used prime editing technology to correct a genetic mutation in the liver cells of a patient with Wilson disease — a rare inherited disorder in which toxic copper accumulates in the body.
The main difference of the method is that DNA can be corrected directly inside the body, without extracting cells, modifying them in the laboratory, and then transplanting them back.
Lipid nanoparticles — a technology similar to that used in mRNA vaccines — were used to deliver the genetic “editor” to the liver.
In the future this could make it possible to treat some inherited diseases with a single procedure instead of lifelong medication.
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