Fig. 10

An overview of the different applications of clustered regularly interspaced palindromic repeat (CRISPR)/CRISPR-associated (Cas) system for in vivo genome editing. A Schematic representation of PEGylated nanoparticles based on the α-helical polypeptide PPABLG (P-HNPs) and their intracellular transport of Cas9 expression plasmid/single guide RNA for genome editing or gene activation. Reprinted from [246] with permission from National Academy of Sciences. B Schematic representation of the LGCP (polyethylene glycol-lipid/gold nanoclusters/Cas9 protein/sgPlk1 plasmid) fabrication process. LGCP delivering Cas9 protein/sgPlk1 plasmid successfully inhibited the progression of the disease by knocking-out the Plk1 gene. Reprinted from [247] with permission from John Wiley & Sons, Inc. C Schematic representation of CRISPR/Cas systems as antiviral therapy: a Cas13 cuts ssRNA and could be used to degrade viral RNA genomes, b inhalable antiviral Cas13a mRNA in rodents, c Hamsters were dosed as indicated with aNLuc mRNA. Lungs were analyzed at 1 d for luminescence, and d lung viral loads from hamsters at 6 d after infection (n = 4). Data represent mean N copy number ± s.e.m. Brown–Forsythe and Welch ANOVA with Dunnett’s multiple comparisons on log-transformed data, where **P = 0.0016 and *P = 0.0198. Reprinted from [248] with permission from Nature