Perspectives, Issues and Solutions in RNAi Therapy: the Expected and the Less Expected

References

• Fire A , XuS , MontgomeryMKet al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature391(6669), 806–811 (1998).
   PubMed Web of Science ®Google Scholar
• Wittrup A , AiA , LiuXet al. Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown. Nat. Biotechnol.33(8), 870–876 (2015).
   PubMed Web of Science ®Google Scholar
• Song E , LeeSK , WangJet al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med.9(3), 347–351 (2003).
   PubMed Web of Science ®Google Scholar
• Tilesi F , FradianiP , SocciVet al. Design and validation of siRNAs and shRNAs. Curr. Opin. Mol. Ther.11(2), 156–164 (2009).
   PubMedGoogle Scholar
• Dixon SJ , StockwellBR. Identifying druggable disease-modifying gene products. Curr. Opin. Chem. Biol.13(5–6), 549–555 (2009).
   PubMed Web of Science ®Google Scholar
• Maurizi A , CapulliM , PatelRet al. RNA interference therapy for autosomal dominant osteopetrosis type 2. Towards the preclinical development. Bone110, 343–354 (2018).
   PubMed Web of Science ®Google Scholar
• Zorde Khvalevsky E , GabaiR , RachmutIHet al. Mutant KRAS is a druggable target for pancreatic cancer. Proc. Natl Acad. Sci. USA110(51), 20723–20728 (2013).
   PubMed Web of Science ®Google Scholar
• Chen D , LiuL , WangKet al. The role of MALAT-1 in the invasion and metastasis of gastric cancer. Scand. J. Gastroenterol.52(6–7), 790–796 (2017).
   PubMed Web of Science ®Google Scholar
• Dong P , XiongY , YueJet al. Long non-coding RNA NEAT1: a novel target for diagnosis and therapy in human tumors. Front. Genet.9, 471 (2018).
   PubMed Web of Science ®Google Scholar
• Kung JT , ColognoriD , LeeJT. Long noncoding RNAs: past, present, and future. Genetics193(3), 651–669 (2013).
   PubMed Web of Science ®Google Scholar
• Bartlett DW , DavisME. Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging. Nucleic Acids Res.34(1), 322–333 (2006).
   PubMed Web of Science ®Google Scholar
• Hoerter JA , KrishnanV , LionbergerTAet al. siRNA-like double-stranded RNAs are specifically protected against degradation in human cell extract. PLoS ONE6(5), e20352039 (2011).
   Web of Science ®Google Scholar
• Dar SA , ThakurA , QureshiAet al. siRNAmod: a database of experimentally validated chemically modified siRNAs. Sci. Rep.6, 20031 (2016).
   PubMed Web of Science ®Google Scholar
• Foster DJ , BrownCR , ShaikhSet al. Advanced siRNA designs further improve in vivo performance of GalNAc-siRNA conjugates. Mol. Ther.26(3), 708–717 (2018).
   PubMed Web of Science ®Google Scholar
• Zhang X , GoelV , AttarwalaHet al. Patisiran pharmacokinetics, pharmacodynamics, and exposure-response analyses in the Phase 3 APOLLO trial in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. J. Clin. Pharmacol.doi: 10.1002/jcph.1480 (2019) ( Epub ahead of print).
   Google Scholar
• Layzer JM , McCaffreyAP , TannerAKet al. In vivo activity of nuclease-resistant siRNAs. RNA10(5), 766–771 (2004).
   PubMed Web of Science ®Google Scholar
• Rajappa M , SaxenaP , KaurJ. Ocular angiogenesis: mechanisms and recent advances in therapy. Adv. Clin. Chem.50, 103–121 (2010).
   PubMed Web of Science ®Google Scholar
• Wicki A , WitzigmannD , BalasubramanianVet al. Nanomedicine in cancer therapy: challenges, opportunities, and clinical applications. J. Control. Release200, 138–157 (2015).
   PubMed Web of Science ®Google Scholar
• Kim B , ParkJH , SailorMJ. Rekindling RNAi therapy: materials design requirements for in vivo siRNA delivery. Adv. Mater.doi: 10.1002/adma.201903637 (2019) ( Epub ahead of print).
   Web of Science ®Google Scholar
• Semple SC , AkincA , ChenJet al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol.28(2), 172–176 (2010).
   PubMed Web of Science ®Google Scholar
• Rajeev KG , NairJK , JayaramanMet al. Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo. Chembiochem16(6), 903–908 (2015).
   PubMed Web of Science ®Google Scholar
• Springer AD , DowdySF. GalNAc-siRNA conjugates: leading the way for delivery of RNAi therapeutics. Nucleic Acid Ther.28(3), 109–118 (2018).
   PubMed Web of Science ®Google Scholar
• Ewe A , PanchalO , PinnapireddySRet al. Liposome-polyethylenimine complexes (DPPC-PEI lipopolyplexes) for therapeutic siRNA delivery in vivo. Nanomedicine13(1), 209–218 (2016).
   PubMed Web of Science ®Google Scholar
• Schafer J , HobelS , BakowskyUet al. Liposome-polyethylenimine complexes for enhanced DNA and siRNA delivery. Biomaterials31(26), 6892–6900 (2010).
   PubMed Web of Science ®Google Scholar
• Kamerkar S , LeBleuVS , SugimotoHet al. Exosomes facilitate therapeutic targeting of oncogenic KRAS in pancreatic cancer. Nature546(7659), 498–503 (2017).
   PubMed Web of Science ®Google Scholar
• Mei Y , WangR , JiangWet al. Recent progress in nanomaterials for nucleic acid delivery in cancer immunotherapy. Biomater. Sci.7(7), 2640–2651 (2019).
   PubMed Web of Science ®Google Scholar
• Feng X , XuW , LiZet al. Immunomodulatory nanosystems. Adv. Sci. (Weinh.)6(17), 1900101 (2019).
   PubMedGoogle Scholar
• Roma-Rodrigues C , MendesR , BaptistaPVet al. Targeting tumor microenvironment for cancer therapy. Int. J. Mol. Sci.20(4), 840 (2019).
   PubMed Web of Science ®Google Scholar
• Yu D , KhanOF , SuvaMLet al. Multiplexed RNAi therapy against brain tumor-initiating cells via lipopolymeric nanoparticle infusion delays glioblastoma progression. Proc. Natl Acad. Sci. USA114(30), E6147–E6156 (2017).
   PubMed Web of Science ®Google Scholar
• Tsouris V , JooMK , KimSHet al. Nano carriers that enable co-delivery of chemotherapy and RNAi agents for treatment of drug-resistant cancers. Biotechnol. Adv.32(5), 1037–1050 (2014).
   PubMed Web of Science ®Google Scholar
• Matsui M , CoreyDR. Non-coding RNAs as drug targets. Nat. Rev. Drug Discov.16(3), 167–179 (2017).
   PubMed Web of Science ®Google Scholar
• Wegner M , DiehlV , BittlVet al. Circular synthesized CRISPR/Cas gRNAs for functional interrogations in the coding and noncoding genome. Elife8, e42549 (2019).
   PubMed Web of Science ®Google Scholar
• Saw PE , SongEW. siRNA therapeutics: a clinical reality. Sci. China Life Sci.doi: 10.1007/s11427-018-9438-y (2019) ( Epub ahead of print).
   Web of Science ®Google Scholar
• Rizk M , TuzmenS. Patisiran for the treatment of patients with familial amyloid polyneuropathy. Drugs Today (Barc.)55(5), 315–327 (2019).
   PubMed Web of Science ®Google Scholar