CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy

@article{Rosenblum2020CRISPRCas9GE,
  title={CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy},
  author={Daniel Rosenblum and Anna Gutkin and Ranit Kedmi and Srinivas Ramishetti and Nuphar Veiga and Ashley M. Jacobi and Mollie S. Schubert and Dinorah Friedmann-Morvinski and Zvi R. Cohen and Mark A. Behlke and Judy Lieberman and Dan Peer},
  journal={Science Advances},
  year={2020},
  volume={6}
}
CRISPR lipid nanoparticles promote in vivo therapeutic genome editing in two aggressive cancer mouse models. Harnessing CRISPR-Cas9 technology for cancer therapeutics has been hampered by low editing efficiency in tumors and potential toxicity of existing delivery systems. Here, we describe a safe and efficient lipid nanoparticle (LNP) for the delivery of Cas9 mRNA and sgRNAs that use a novel amino-ionizable lipid. A single intracerebral injection of CRISPR-LNPs against PLK1 (sgPLK1-cLNPs) into… 

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