Potential use of glucuronylglucosyl-β-cyclodextrin/dendrimer conjugate (G2) as a siRNA carrier for the treatment of familial amyloidotic polyneuropathy

References

• Wang J, Lu Z, Wientjes MG, Au JL. Delivery of siRNA therapeutics: barriers and carriers. AAPS J 2010;12:492–503
   PubMed Web of Science ®Google Scholar
• Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference-based therapeutics. Nature 2009;457:426–33
   PubMed Web of Science ®Google Scholar
• Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat Rev Genet 2007;8:173–84
   PubMed Web of Science ®Google Scholar
• Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by doublestranded RNA in Caenorhabditis elegans. Nature 1998;391:806–11
   PubMed Web of Science ®Google Scholar
• Benson MD, Uemichi T. Transthyretin amyloidosis. Amyloid 1996;3:44–56
   Web of Science ®Google Scholar
• Ando Y, Nakamura M, Araki S. Transthyretin-related familial amyloidotic polyneuropathy. Arch Neurol 2005;62:1057–62
   PubMed Web of Science ®Google Scholar
• Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve 2007;14:277–82
   Google Scholar
• Connors LH, Lim A, Prokaeva T, et al. Tabulation of human transthyretin (TTR) variants. Amyloid 2003;10:160–84
   PubMed Web of Science ®Google Scholar
• Westermark P, Benson MD, Buxbaum JN. Amyloid fibril protein nomenclature-2002. Amyloid 2002;99:197–200
   Google Scholar
• Ando Y. Liver transplantation and new therapeutic approaches for familial amyloidotic polyneuropathy (FAP). Med Mol Morphol 2005;38:142–54
   PubMedGoogle Scholar
• Ando Y, Tanaka Y, Ando E, et al. Effect of liver transplantation on autonomic dysfunction in familial amyloidotic polyneuropathy type I. Lancet 1995;354:195–6
   Web of Science ®Google Scholar
• Holmgren G, Steen L, Suhr O, et al. Clinical improvement and amyloid regression after liver transplantation in hereditary transthretin amyloidosis. Lancet 1993;341:1113–6
   PubMed Web of Science ®Google Scholar
• Benson MD, Kluve-Beckerman B, Zeldenrust SR, et al. Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides. Muscle Nerve 2006;33:609–18
   PubMed Web of Science ®Google Scholar
• Semple SC, Akinc A, Chen J, et al. Rational design of cationic lipids for siRNA delivery. Nat Biotechnol 2010;28:172–6
   PubMed Web of Science ®Google Scholar
• Song E, Lee SK, Wang J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003;9:347–51
   PubMed Web of Science ®Google Scholar
• Zhou J, Rossi JJ. Aptamer-targeted cell-specific RNA interference. Silence 2010;1:4 . doi: 10.1186/1758-907X-1-4
   PubMedGoogle Scholar
• Arima H, Kihara F, Hirayama F, Uekama K. Enhancement of gene expression by polyamidoamine dendrimer conjugates with α-, β-, and γ-cyclodextrins. Bioconjug Chem 2001;12:476–84
   PubMed Web of Science ®Google Scholar
• Kihara F, Arima H, Tsutsumi T, et al. Effects of structure of polyamidoamine dendrimer on gene transfer efficiency of the dendrimer conjugate with α-cyclodextrin. Bioconjug Chem 2002;13:1211–19
   PubMed Web of Science ®Google Scholar
• Kihara F, Arima H, Tsutsumi T, et al. In vitro and in vivo gene transfer by an optimized α-cyclodextrin conjugate with polyamidoamine dendrimer. Bioconjug Chem 2003;14:342–50
   PubMed Web of Science ®Google Scholar
• Arima H, Motoyama K, Higashi T. Sugar-appended polyamidoamine dendrimer conjugates with cyclodextrins as cell-specific non-viral vectors. Adv Drug Deliv Rev 2013;65:1204–14
   PubMed Web of Science ®Google Scholar
• Arima H, Motoyama K. Recent findings concerning PAMAM dendrimer conjugates with cyclodextrins as carriers of DNA and RNA. Sensors 2009;9:6346–61
   PubMed Web of Science ®Google Scholar
• Arima H, Motoyama K, Higashi T. Polyamidoamine dendrimer conjugates with cyclodextrins as novel carriers for DNA, shRNA and siRNA. Pharmaceutics 2012;4:130–48
   PubMedGoogle Scholar
• Arima H, Motoyama K, Higashi T. Cyclodextrin/dendrimer conjugates as DNA and oligonucleotide carriers. Curr Top Med Chem 2014;14:465–77
   PubMed Web of Science ®Google Scholar
• Tsutsumi T, Hirayama F, Uekama K, Arima H. Evaluation of polyamidoamine dendrimer/α-cyclodextrin conjugate (generation 3, G3) as a novel carrier for small interfering RNA (siRNA). J Control Release 2007;119:349–59
   PubMed Web of Science ®Google Scholar
• Arima H, Tsutsumi T, Yoshimatsu A, et al. Inhibitory effect of siRNA complexes with polyamidoamine dendrimer/α-cyclodextrin conjugate (generation 3, G3) on endogenous gene expression. Eur J Pharm Sci 2011;44:375–84
   PubMed Web of Science ®Google Scholar
• Arima H, Motoyama K, Higashi T. Potential use of polyamidoamine dendrimer conjugates with cyclodextrins as novel carriers for siRNA. Pharmaceuticals 2011;5:61–78
   Google Scholar
• Hayashi Y, Mori Y, Yamashita S, et al. Potential use of lactosylated dendrimer (G3)/α-cyclodextrin conjugates as hepatocyte-specific siRNA carriers for the treatment of familial amyloidotic polyneuropathy. Mol Pharm 2012;9:1645–53
   PubMed Web of Science ®Google Scholar
• Anno T, Motoyama K, Higashi T, et al. Preparation and evaluation polyamidoamine dendrimer (G2)/branched-β-cyclodextrin conjugate as a novel gene transfer carrier. J Incl Phenom Macrocycl Chem 2011;70:339–44
   Web of Science ®Google Scholar
• Anno T, Higashi T, Motoyama K, et al. Potential use of glucuronylglucosyl-β-cyclodextrin/dendrimer conjugate (G2) as a DNA carrier in vitro and in vivo. J Drug Target 2012;20:272–80
   PubMed Web of Science ®Google Scholar
• Anno T, Higashi T, Motoyama K, et al. Possible enhancing mechanisms for gene transfer activity of glucuronylglucosyl-β-cyclodextrin/dendrimer conjugate. Int J Pharm 2012;426:239–47
   PubMed Web of Science ®Google Scholar
• Arima H, Yoshimatsu A, Ikeda H, et al. Folate-PEG-appended dendrimer conjugate with α-cyclodextrin as a novel cancer cell-selective siRNA delivery carrier. Mol Pharm 2012;9:2591–604
   PubMed Web of Science ®Google Scholar
• Arima H, Yamashita S, Mori Y, et al. In vitro and in vivo gene delivery mediated by Lactosylated dendrimer/α-cyclodextrin conjugates (G2) into hepatocytes. J Control Release 2010;146:106–17
   PubMed Web of Science ®Google Scholar
• Hayashi Y, Higashi T, Motoyama K, et al. Design and evaluation of polyamidoamine dendrimer conjugate with PEG, α-cyclodextrin and lactose as a novel hepatocyte-selective gene carrier in vitro and in vivo. J Drug Target 2013;21:487–96
   PubMed Web of Science ®Google Scholar
• Uehata K, Anno T, Hayashida K, et al. Peak-less hypoglycemic effect of insulin glargine by complexation with maltosyl-β-cyclodextrin. Int J Pharm 2012;422:33–9
   PubMed Web of Science ®Google Scholar
• Tavornvipas S, Tajiri S, Hirayama F, et al. Effects of hydrophilic cyclodextrins on aggregation of recombinant human growth hormone. Pharm Res 2004;21:2369–76
   PubMed Web of Science ®Google Scholar
• Tavornvipas S, Hirayama F, Takeda S, et al. Effects of cyclodextrins on chemically and thermally induced unfolding and aggregation of lysozyme and basic fibroblast growth factor. J Pharm Sci 2006;95:2722–9
   PubMed Web of Science ®Google Scholar
• Irie T, Uekama K. Cyclodextrins in peptide and protein delivery. Adv Drug Deliv Rev 1999;36:101–23
   PubMed Web of Science ®Google Scholar
• Jono H, Anno T, Motoyama K, et al. Cyclodextrin, a novel therapeutic tool for suppressing amyloidogenic transthyretin misfolding in transthyretin-related amyloidosis. Biochem J 2011;437:35–42
   PubMed Web of Science ®Google Scholar