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CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein. When the researchers tested CRISPRoff’s ability to silence the prion protein gene, they found that it was effective and stable.
Now, his focus is on Gylden Pharma’s nanoparticle-based vaccine platform – a cutting-edge solution that bypasses the limitations of traditional RNA and live attenuated virus vaccines. Human trials and real-world efficacy Gyldens platform has also shown promising results in early-stage human trials.
Although Amodei does acknowledge some real-world issues limiting scientific progress — such as the slow growth of organisms and tedious clinical trials — he mostly passes over the more general tools that will be required to accelerate research in the near term. This essay focuses on how we might do both, specifically for the cell.
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