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So the actual tech here is: * mRNA and lipid nano particles to get the mRNA into cells * 2 pieces of mRNA. One codes for the change to break the gene for the
by LatteLazy 5y ago
So the actual tech here is:
* mRNA and lipid nano particles to get the mRNA into cells
* 2 pieces of mRNA. One codes for the change to break the gene for the protein being targeted and another to create a enzyme to edit the first piece into the genome
* both bits of mRNA need to be in the cell, then the enzyme and gene are created and the enzyme edits in the gene
So any cell where this works correctly (and its descendents) no longer produces the protein. It appears that about 60% of cells get hit successfully (based on a 60% drop on the protein level).
They only targeted liver cells. That's good because the liver tends to soak up foreign materials from blood. But edits were found at low levels in other organs (spleen etc). That shouldn't make much difference as the protein in question is only produced in the liver. But beware I guess as a multi-use protein could be altered in multiple organs.
Also, it looks like the edit was very accurate and didn't break other genes at anything like the rate it broke the target.
This is pretty incredible stuff. The biochemical equivalent of keyhole surgery.