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The bulk of my nearly three decades of experience up to that point was with drugging protein targets using a variety of modalities, but principally smallmolecules. PEARL-seq gives us insight into RNA engagement in the cell, but not functional outcomes. Prior to 2015, I had a casual relationship, at best, with targeting RNA.
Read Inhalable nanoparticles, packaged with mRNA or CRISPR systems, efficiently edit lung cells. They achieved high gene-editing efficiencies in airway epithelial cells (which are normally hard to target.) Read A really simple way to isolate AAVs, which are a type of virus often used to deliver gene therapies into cells.
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