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10.1016/j.addr.2020.06.026

http://scihub22266oqcxt.onion/10.1016/j.addr.2020.06.026
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32622021!7329694!32622021
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suck abstract from ncbi


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pmid32622021      Adv+Drug+Deliv+Rev 2020 ; 159 (ä): 344-363
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  • Lipid nanoparticle technology for therapeutic gene regulation in the liver #MMPMID32622021
  • Witzigmann D; Kulkarni JA; Leung J; Chen S; Cullis PR; van der Meel R
  • Adv Drug Deliv Rev 2020[]; 159 (ä): 344-363 PMID32622021show ga
  • Hereditary genetic disorders, cancer, and infectious diseases of the liver affect millions of people around the globe and are a major public health burden. Most contemporary treatments offer limited relief as they generally aim to alleviate disease symptoms. Targeting the root cause of diseases originating in the liver by regulating malfunctioning genes with nucleic acid-based drugs holds great promise as a therapeutic approach. However, employing nucleic acid therapeutics in vivo is challenging due to their unfavorable characteristics. Lipid nanoparticle (LNP) delivery technology is a revolutionary development that has enabled clinical translation of gene therapies. LNPs can deliver siRNA, mRNA, DNA, or gene-editing complexes, providing opportunities to treat hepatic diseases by silencing pathogenic genes, expressing therapeutic proteins, or correcting genetic defects. Here we discuss the state-of-the-art LNP technology for hepatic gene therapy including formulation design parameters, production methods, preclinical development and clinical translation.
  • |*Gene Expression Regulation[MESH]
  • |*Gene Transfer Techniques[MESH]
  • |*Genetic Therapy[MESH]
  • |Animals[MESH]
  • |Humans[MESH]
  • |Lipids/*administration & dosage[MESH]
  • |Liver/*metabolism[MESH]
  • |Nanoparticles/*administration & dosage[MESH]


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