Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

  title={Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.},
  author={Akin Akinc and William Querbes and Soma De and June Qin and Maria Frank-Kamenetsky and K. Narayanannair Jayaprakash and M. Jayaraman and Kallanthottathil G Rajeev and William L Cantley and Joseph Robert Dorkin and James S. Butler and Liuliang Qin and Timothy S Racie and Andrew G. Sprague and Eugenio Fava and Anja Zeigerer and Michael J. Hope and Marino Zerial and Dinah W. Y. Sah and Kevin Fitzgerald and Mark A. Tracy and Muthiah Manoharan and Victor Koteliansky and Antonin R. de Fougerolles and Martin A. Maier},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={18 7},
Lipid nanoparticles (LNPs) have proven to be highly efficient carriers of short-interfering RNAs (siRNAs) to hepatocytes in vivo; however, the precise mechanism by which this efficient delivery occurs has yet to be elucidated. We found that apolipoprotein E (apoE), which plays a major role in the clearance and hepatocellular uptake of physiological lipoproteins, also acts as an endogenous targeting ligand for ionizable LNPs (iLNPs), but not cationic LNPs (cLNPs). The role of apoE was… CONTINUE READING
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