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Review

Advances with RNA interference in Alzheimer’s disease research

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Pages 117-125 | Published online: 22 Feb 2013

References

  • Alzheimer A Über eine eigenartige Erkrankung der Hirnrinde [Concerning a novel disease of the cortex] Allgemeine Zeitschrift fur Psychiatrie und Psychisch-gerichtliche Medizin 1907 64 146 148 German
  • Blennow K de Leon MJ Zetterberg H Alzheimer’s disease Lancet 2006 368 9533 387 403 16876668
  • Citron M Alzheimer’s disease: strategies for disease modification Nat Rev Drug Discov 2010 9 5 387 398 20431570
  • Glenner GG Murphy MA Amyloidosis of the nervous system J Neurol Sci 1989 94 1–3 1 28 2559163
  • Goedert M Wischik CM Crowther RA Walker JE Klug A Cloning and sequencing of the cDNA encoding a core protein of the paired helical filament of Alzheimer disease: identification as the microtubule-associated protein tau Proc Natl Acad Sci U S A 1988 85 11 4051 4055 3131773
  • Goedert M Spillantini MG Jakes R Rutherford D Crowther RA Multiple isoforms of human microtubule-associated protein tau: sequences and localization in neurofibrillary tangles of Alzheimer’s disease Neuron 1989 3 4 519 526 2484340
  • Hardy JA Selkoe DJ The amyloid hypothesis of Alzheimer’s disease: progress and problems on the road to therapeutics Science 2002 297 5580 353 356 12130773
  • Hardy J Alzheimer’s disease: the amyloid cascade hypothesis: an update and reappraisal J Alzheimers Dis 2006 9 Suppl 3 151 153 16914853
  • Golde TE Petrucelli L Lewis J Targeting Abeta and tau in Alzheimer’s disease, an early interim report Exp Neurol 2010 223 2 252 266 19716367
  • Fire A Xu S Montgomery MK Kostas ASA Driver SE Mello CC Potent and specifIc genetic interference by double-stranded RNA in Caenorhabditis elegans Nature 1998 391 6669 806 811 9486653
  • Daneholt B Advanced information: RNA interference. Nobelprize.org [website on Internet]. 2012 Available from: http://www.nobelprize.org/nobel_prizes/medicine/laureates/2006/advanced.html AccessedNovember 8, 2012
  • Seyhan AA Rya TE RNAi screening for the discovery of novel modulators of human disease Curr Pharm Biotechnol 2010 11 7 735 756 20420566
  • Davidson BL Boudreau RL RNA interference: a tool for querying nervous system function and an emerging therapy Neuron 2007 53 6 781 788 17359914
  • Gonzalez-Alegre P Paulson HL Technology insight: therapeutic RNA interference – how far from the neurology clinic? Nat Clin Pract Neurol 2007 3 7 394 404 17611488
  • Lingor P Bähr M Targeting neurological disease with RNAi Mol Biosyst 2007 3 11 773 780 17940660
  • Perrimon N Ni JQ Perkins L In vivo RNAi: today and tomorrow Cold Spring Harb Perspect Biol 2010 2 8 a003640 20534712
  • Priller C Bauer T Mitteregger G Krebs B Kretzschmar HA Herms J Synapse formation and function is modulated by the amyloid precursor protein J Neurosci 2006 26 27 7212 7221 16822978
  • Turner PR O’Connor K Tate WP Abraham WC Roles of amyloid precursor protein and its fragments in regulating neural activity, plasticity and memory Prog Neurobiol 2003 70 1 32 12927332
  • Duce JA Tsatsanis A Cater MA Iron-export ferroxidase activity of â-amyloid precursor protein is inhibited by zinc in Alzheimer’s disease Cell 2010 142 6 857 867 20817278
  • Zheng H Koo EH The amyloid precursor protein: beyond amyloid Mol Neurodegener 2006 1 5 16930452
  • Forman MS Trojanowski JQ Lee VM Neurodegenerative diseases: a decade of discoveries paves the way for therapeutic breakthroughs Nat Med 2004 10 10 1055 1063 15459709
  • LaFerla FM Green KN Oddo S Intracellular amyloid-beta in Alzheimer’s disease Nat Rev Neurosci 2007 8 7 499 509 17551515
  • Chyung AS Greenberg BD Cook DG Doms RW Lee VM Novel beta-secretase cleavage of beta-amyloid precursor protein in the endoplasmic reticulum/intermediate compartment of NT2N cells J Cell Biol 1997 138 3 671 680 9245794
  • Xu H Sweeney D Wang R Generation of Alzheimer beta-amyloid protein in the trans-Golgi network in the apparent absence of vesicle formation Proc Natl Acad Sci U S A 1997 94 8 3748 3752 9108049
  • Koo EH Squazzo SL Evidence that production and release of amyloid beta-protein involves the endocytic pathway J Biol Chem 1994 269 26 17386 17389 8021238
  • Capell A Steiner H Willem M Maturation and pro-peptide cleavage of beta-secretase J Biol Chem 2000 275 40 30849 30854 10801872
  • Huse JT Pijak DS Leslie GJ Lee VM Doms RW Maturation and endosomal targeting of beta-site amyloid precursor protein-cleaving enzyme. The Alzheimer’s disease beta-secretase J Biol Chem 2000 275 43 33729 33737 10924510
  • Bennett BD Denis P Haniu M A furin-like convertase mediates propeptide cleavage of BACE, the Alzheimer’s beta-secretase J Biol Chem 2000 275 48 33712 33717 10922360
  • Creemers JW Ines Dominguez D Plets E Processing of beta-secretase by furin and other members of the proprotein convertase family J Biol Chem 2001 276 6 4211 4217 11071887
  • Koh YH von Arnim CA Hyman BT Tanzi RE Tesco G BACE is degraded via the lysosomal pathway J Biol Chem 2005 280 37 32499 32504 16033761
  • Walter J Fluhrer R Hartung B Phosphorylation regulates intracellular trafficking of beta-secretase J Biol Chem 2001 276 18 14634 14641 11278841
  • Vassar R Bennett BD Babu-Khan S Beta-secretase cleavage of Alzheimer’s amyloid precursor protein by the transmembrane aspartic protease BACE Science 1999 286 5440 735 741 10531052
  • Choy RW Cheng Z Schekman R Amyloid precursor protein (APP) traffics from the cell surface via endosomes for amyloid â (Aâ) production in the trans-Golgi network Proc Natl Acad Sci U S A 2012 109 30 E2077 E2082 22711829
  • Finan GM Okada H Kim TW BACE1 retrograde trafficking is uniquely regulated by the cytoplasmic domain of sortilin J Biol Chem 2011 286 14 12602 12616 21245145
  • Hoyer S Glucose metabolism and insulin receptor signal transduction in Alzheimer disease Eur J Pharmacol 2004 490 1–3 115 125 15094078
  • Zou L Wang Z Shen L Receptor tyrosine kinases positively regulate BACE activity and Amyloid-beta production through enhancing BACE internalization Cell Res 2007 17 5 389 401 17325690
  • Bonifacino JS The GGA proteins: adaptors on the move Nat Rev Mol Cell Biol 2004 5 1 33 32 14708008
  • Wahle T Prager K Raffler N Haass C Famulok M Walter J GGA proteins regulate retrograde transport of BACE1 from endosomes to the trans-Golgi network Mol Cell Neurosci 2005 29 3 453 461 15886016
  • He X Li F Chang WP Tang J GGA proteins mediate the recycling pathway of memapsin 2 (BACE) J Biol Chem 2005 280 12 11696 11703 15615712
  • Tesco G Koh YH Kang EL Depletion of GGA3 stabilizes BACE and enhances beta-secretase activity Neuron 2007 54 5 721 737 17553422
  • Cuervo AM Autophagy: in sickness and in health Trends Cell Biol 2004 14 2 70 77 15102438
  • Chu CT Autophagic stress in neuronal injury and disease J Neuropathol Exp Neurol 2006 65 5 423 432 16772866
  • Yu WH Kumar A Peterhoff C Autophagic vacuoles are enriched in amyloid precursor protein-secretase activities: implications for beta-amyloid peptide over-production and localization in Alzheimer’s disease Int J Biochem Cell Biol 2004 36 12 2531 2540 15325590
  • Yu WH Cuervo AM Kumar A Macroautophagy – a novel Betaamyloid peptide-generating pathway activated in Alzheimer’s disease J Cell Biol 2005 171 1 87 98 16203860
  • Florez-McClure ML Hohsfield LA Fonte G Bealor MT Link CD Decreased insulin-receptor signaling promotes the autophagic degradation of beta-amyloid peptide in C. elegans Autophagy 2007 3 6 569 580 17675890
  • Hornsten A Lieberthal J Fadia S APL-1, a Caenorhabditis elegans protein related to the human beta-amyloid precursor protein, is essential for viability Proc Natl Acad Sci U S A 2007 104 6 1971 1976 17267616
  • Wiese M Antebi A Zheng H Intracellular trafficking and synaptic function of APL-1 in Caenorhabditis elegans PLoS One 2010 5 9 pii:e12790
  • Fahrenholz F Alpha-secretase as a therapeutic target Curr Alzheimer Res 2007 4 4 412 417 17908044
  • Allinson TM Parkin ET Turner AJ Hooper NM ADAMs family members as amyloid precursor protein alpha-secretases J Neurosci Res 2003 74 3 342 352 14598310
  • Kuhn PH Wang H Dislich B ADAM10 is the physiologically relevant, constitutive alpha-secretase of the amyloid precursor protein in primary neurons Embo J 2010 29 17 3020 3032 20676056
  • Dando PM Brown MA Barrett AJ Human thimet oligopeptidase Biochem J 1993 294 Pt 2 451 457 8373360
  • Knight CG Dando PM Barrett AJ Thimet oligopeptidase specificity: evidence of preferential cleavage near the C-terminus and product inhibition from kinetic analysis of peptide hydrolysis Biochem J 1995 308 Pt 1 145 150 7755557
  • Shrimpton CN Smith AI Soluble neutral metallopeptidases: physiological regulators of peptide action J Pept Sci 2000 6 6 251 263 10912905
  • Shrimpton CN Smith AI Lew RA Soluble metalloendopeptidases and neuroendocrine signaling Endocr Rev 2002 23 5 647 664 12372844
  • Koike H Seki H Kouchi Z Thimet oligopeptidase cleaves the full-length Alzheimer amyloid precursor protein at a beta-secretase cleavage site in COS cells J Biochem 1999 126 1 235 242 10393344
  • Yamin R Malgeri EG Sloane JA McGraw WT Abraham CR Metalloendopeptidase EC 3.4.24.15 is necessary for Alzheimer’s amyloidbeta peptide degradation J Biol Chem 1999 274 26 18777 18784 10373494
  • Pollio G Hoozemans JJ Andersen CA Increased expression of the oligopeptidase THOP1 is a neuroprotective response to Abeta toxicity Neurobiol Dis 2008 31 1 145 158 18571100
  • Puglielli L Konopka G Pack-Chung E Acyl-coenzyme A: cholesterol acyltransferase modulates the generation of the amyloid beta-peptide Nat Cell Biol 2001 3 10 905 912 11584272
  • Hutter-Paier B Huttunen HJ Puglielli The ACAT inhibitor CP-113,818 markedly reduces amyloid pathology in a mouse model of Alzheimer’s disease Neuron 2004 44 2 227 238 15473963
  • Huttunen HJ Greco G Kovacs DM Knockdown of ACAT-1 reduces amyloidogenic processing of APP FEBS Lett 2007 581 8 1688 1692 17412327
  • Liu B Moloney A Meehan S Iron promotes the toxicity of amyloid beta peptide by impeding its ordered aggregation J Biol Chem 2011 286 6 4248 4256 21147772
  • Weingarten MD Lockwood AH Hwo SY Kirschner MW A protein factor essential for microtubule assembly Proc Natl Acad Sci U S A 1975 72 5 1858 1862 1057175
  • Alonso A Zaidi T Novak M Grundke-Iqbal I Iqbal K Hyperphosphorylation induces self-assembly of tau into tangles of paired helical filaments/straight filaments Proc Natl Acad Sci U S A 2001 98 12 6923 6928 11381127
  • Guthrie CR Schellenberg GD Kraemer BC SUT-2 potentiates tauinduced neurotoxicity in Caenorhabditis elegans Hum Mol Genet 2009 18 10 1825 1838 19273536
  • Guthrie CR Greenup L Leverenz JB Kraemer BC MSUT2 is a determinant of susceptibility to tau neurotoxicity Hum Mol Genet 2011 20 10 1989 1999 21355046
  • Baumann K Mandelkow EM Biernat J Piwnica-Worms H Mandelkow E Abnormal Alzheimer-like phosphorylation of tau-protein by cyclin-dependent kinases cdk2 and cdk5 FEBS Lett 1993 336 3 417 424 8282104
  • Paudel HK Lew J Ali Z Wang JH Brain proline-directed protein kinase phosphorylates tau on sites that are abnormally phosphorylated in tau associated with Alzheimer’s paired helical filaments J Biol Chem 1993 268 31 23512 23518 8226879
  • Piedrahita D Hernández I López-Tobón A Silencing of CDK5 reduces neurofibrillary tangles in transgenic alzheimer’s mice J Neurosci 2010 30 42 13966 13976 20962218
  • Haass C Selkoe DJ Soluble protein oligomers in neurodegeneration: lessons from the Alzheimer’s amyloid beta-peptide Nat Rev Mol Cell Biol 2007 8 2 101 112 17245412
  • Shankar GM Li S Mehta TH Amyloid-beta protein dimers isolated directly from Alzheimer’s brains impair synaptic plasticity and memory Nat Med 2008 14 8 837 842 18568035
  • Jin M Shepardson N Yang T Chen G Walsh D Selkoe DJ Soluble amyloid beta-protein dimers isolated from Alzheimer cortex directly induce Tau hyperphosphorylation and neuritic degeneration Proc Natl Acad Sci U S A 2011 108 14 5819 5824 21421841
  • Schmetsdorf S Arnold E Holzer M Arendt T Gärtner U A putative role for cell cycle-related proteins in microtubule-based neuroplasticity Eur J Neurosci 2009 29 6 1096 1107 19302146
  • Heldin CH Miyazono K ten Dijke P TGF-beta signalling from cell membrane to nucleus through SMAD proteins Nature 1997 390 6659 465 471 9393997
  • Ueberham U Ueberham E Gruschka H Arendt T Altered subcellular location of phosphorylated Smads in Alzheimer’s disease Eur J Neurosci 2006 24 8 2327 2334 17074053
  • Ueberham U Hilbrich I Ueberham E Transcriptional control of cell cycle-dependent kinase 4 by Smad proteins – implications for Alzheimer’s disease Neurobiol Aging 2012 33 12 2827 2840 22418736
  • Song B Davis K Liu XS Lee HG Smith M Liu X Inhibition of Polo-like kinase 1 reduces beta-amyloid-induced neuronal cell death in Alzheimer’s disease Aging (Albany N Y) 2011 3 9 846 851
  • Wiese M Antebi A Zheng H Regulation of neuronal APL-1 expression by cholesterol starvation PLoS One 2012 7 2 e32038 22363792
  • Borchelt DR Thinakaran G Eckman CB Familial Alzheimer’s disease-linked presenilin 1 variants elevate Abeta1-42/1-40 ratio in vitro and in vivo Neuron 1996 17 5 1005 1013 8938131
  • Duff K Eckman C Zehr C Increased amyloid-beta42(43) in brains of mice expressing mutant presenilin 1 Nature 1996 383 6602 710 713 8878479
  • Bali J Gheinani AH Zurbriggen S Rajendran L Role of genes linked to sporadic Alzheimer’s disease risk in the production of â-amyloid peptides Proc Natl Acad Sci U S A 2012 109 38 15307 15311 22949636
  • Trimmer PA Swerdlow RH Parks JK Abnormal mitochondrial morphology in sporadic Parkinson’s and Alzheimer’s disease cybrid cell lines Exp Neurol 2000 162 1 37 50 10716887
  • Shin HW Shinotsuka C Torii S Murakami K Nakayama K Identification and subcellular localization of a novel mammalian dynamin-related protein homologous to yeast Vps1p and Dnm1p J Biochem 1997 122 3 525 530 9348079
  • Wang X Su B Fujioka H Zhu X Dynamin-like protein 1 reduction underlies mitochondrial morphology and distribution abnormalities in fibroblasts from sporadic Alzheimer’s disease patients Am J Pathol 2008 173 2 470 482 18599615
  • Burnett JC Rossi JJ Tiemann K Current progress of siRNA/shRNA therapeutics in clinical trials Biotechnol J 2011 6 9 1130 1146 21744502
  • Burnett JC Rossi JJ RNA-based therapeutics: current progress and future prospects Chem Biol 2012 19 1 60 71 22284355
  • Rodriguez-Lebron E Paulson HL Allele-specific RNA interference for neurological disease Gene Ther 2006 13 6 576 581 16355113
  • Rodríguez-Lebrón E Gouvion CM Moore SA Davidson BL Paulson HL Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer’s disease Mol Ther 2009 17 9 1563 1573 19532137
  • Alvarez-Erviti L Seow Y Yin H Betts C Lakhal S Wood MJ Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes Nat Biotechnol 2011 29 4 341 345 21423189
  • Lublin A Isoda F Patel H FDA-approved drugs that protect mammalian neurons from glucose toxicity slow aging dependent on cbp and protect against proteotoxicity PLoS One 2011 6 11 e27762 22114686
  • Azorsa DO Robeson RH Frost D High-content siRNA screening of the kinome identifies kinases involved in Alzheimer’s disease-related tau hyperphosphorylation BMC Genomics 2010 11 25 20067632
  • Lambert JC Amouyel P Genetic heterogeneity of Alzheimer’s disease: complexity and advances Psychoneuroendocrinology 2007 32 Suppl 1 S62 S70 17659844
  • Whitehead KA Langer R Anderson DG Knocking down barriers: advances in siRNA delivery Nat Rev Drug Discov 2009 8 2 129 138 19180106
  • Alzheimer’s Association Alzheimer’s disease facts and figures Alzheimers Dement 2012 8 2 131 168 22404854
  • Gomase VS Tagore S RNAi – a tool for target finding in new drug development Curr Drug Metab 2008 9 3 241 244 18336228
  • Seyhan AA RNAi: a potential new class of therapeutic for human genetic disease Hum Genet 2011 130 5 583 605 21537948
  • Bumcrot D Manoharan M Koteliansky V Sah DW RNAi therapeutics: a potential new class of pharmaceutical drugs Nat Chem Biol 2006 2 12 711 719 17108989
  • Brunden KR Trojanowski JQ Lee VM Advances in tau-focused drug discovery for Alzheimer’s disease and related tauopathies Nat Rev Drug Discov 2009 8 10 783 793 19794442