07/15/2020 12:00 AM EDT
Source: National Institutes of Health (NIH). Published: 7/15/2020. The speaker in this one-hour lecture describes his work on the combinatorial development of nanoparticulate, intracellular delivery systems for RNA therapy and gene editing. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated, and screened for their ability to deliver macromolecular payloads inside of cells. These nanoformulations facilitate in vivo delivery, enabling gene suppression with small-interfering RNA, gene expression with messenger RNA, or permanent genetic editing using the CRISPR/Cas9 system. (Video or Multimedia)
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