Nature published a brief summary of the recent CRISPR treatment in vivo for a rare disease. They note:
Intellia encased RNA molecules coding the guide RNA and the Cas9 protein in nanoparticles made of biomolecules called lipids, which can be taken up by the liver. The guide RNA sends Cas9 to snip the TTR gene; the cell’s DNA-repair processes then mend the break, but often make mistakes that can disable the gene and halt production of the TTR protein. A month after treatment, TTR production was reduced in all participants. In one person who received the higher of the two tested doses, it fell by 96% — a result that Gillmore, who was an investigator on the trial, finds particularly exciting. Current treatments can reduce TTR by as much as 80%. But he says that a therapy that knocks TTR production down further would increase the chance that his patients’ symptoms would stop getting worse — and might even lead to improvement.
Sound familiar, it is mRNA in a lipid carrier. just like the COVID vaccine. But now it targets certain cells producing the missing protein.
We recently published a paper on how this can be used with prostate cancer targeting the PSMA surface protein of the malignant cells and using CRISPR to enter the cell and stop its proliferation through a forced apoptosis. Just a thought piece but wait a year or two.
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