Novel regulators of PrP^C expression as potential therapeutic targets in prion diseases

References

• Caughey B, Chesebro B. Prion protein and the transmissible spongiform encephalopathies. Trends Cell Biol. 1997 Feb;7(2):56–62. PubMed PMID: 17708907.
   PubMed Web of Science ®Google Scholar
• Colby DW, Prusiner SB. Prions. Cold Spring Harb Perspect Biol. 2011 Jan 1;3(1):a006833. PubMed PMID: 21421910; PubMed Central PMCID: PMCPMC3003464.
   PubMed Web of Science ®Google Scholar
• Prusiner SB. Novel proteinaceous infectious particles cause scrapie. Science. 1982 Apr 9;216(4542):136–144. PubMed PMID: 6801762.
   PubMed Web of Science ®Google Scholar
• Weissmann C, Enari M, Klohn PC, et al. Transmission of prions. Proc Natl Acad Sci U S A. 2002 Dec 10;99(Suppl 4):16378–16383. PubMed PMID: 12181490; PubMed Central PMCID: PMCPMC139897.
   PubMed Web of Science ®Google Scholar
• Prusiner SB. Prions. Proc Natl Acad Sci U S A. 1998 Nov 10;95(23):13363–13383. PubMed PMID: 9811807; PubMed Central PMCID: PMCPMC33918.
   PubMed Web of Science ®Google Scholar
• Prusiner SB. Molecular biology of prion diseases. Science. 1991 Jun 14;252(5012):1515–1522. PubMed PMID: 1675487.
   PubMed Web of Science ®Google Scholar
• Bieschke J, Weber P, Sarafoff N, et al. Autocatalytic self-propagation of misfolded prion protein. Proc Natl Acad Sci U S A. 2004 Aug 17;101(33):12207–12211. PubMed PMID: 15297610; PubMed Central PMCID: PMCPMC514458.
   PubMed Web of Science ®Google Scholar
• Giese A, Kretzschmar HA. Prion-induced neuronal damage–the mechanisms of neuronal destruction in the subacute spongiform encephalopathies. Curr Top Microbiol Immunol. 2001;253:203–217. PubMed PMID: 11417136.
   PubMed Web of Science ®Google Scholar
• Brandner S, Isenmann S, Raeber A, et al. Normal host prion protein necessary for scrapie-induced neurotoxicity. Nature. 1996 Jan 25;379(6563):339–343. PubMed PMID: 8552188.
   PubMed Web of Science ®Google Scholar
• Alibhai J, Blanco RA, Barria MA, et al. Distribution of misfolded prion protein seeding activity alone does not predict regions of neurodegeneration. PLoS Biol. 2016 Nov;14(11):e1002579. PubMed PMID: 27880767; PubMed Central PMCID: PMCPMC5120774.
   PubMed Web of Science ®Google Scholar
• Prusiner SB. Biology and genetics of prions causing neurodegeneration. Annu Rev Genet. 2013;47(1):601–623. PubMed PMID: 24274755; PubMed Central PMCID: PMCPMC4010318.
   PubMed Web of Science ®Google Scholar
• Kuwata K, Nishida N, Matsumoto T, et al. Hot spots in prion protein for pathogenic conversion. Proc Natl Acad Sci U S A. 2007 Jul 17;104(29):11921–11926. PubMed PMID: 17616582; PubMed Central PMCID: PMCPMC1924567.
   PubMed Web of Science ®Google Scholar
• Aguzzi A, Glatzel M, Montrasio F, et al. Interventional strategies against prion diseases. Nat Rev Neurosci. 2001 Oct;2(10):745–749. PubMed PMID: 11584312.
   PubMed Web of Science ®Google Scholar
• Pankiewicz JE, Sanchez S, Kirshenbaum K, et al. Anti-prion protein antibody 6D11 restores cellular proteostasis of prion protein through disrupting recycling propagation of PrP(Sc) and targeting PrP(Sc) for lysosomal degradation. Mol Neurobiol. 2019 Mar;56(3):2073–2091. PubMed PMID: 29987703; PubMed Central PMCID: PMCPMC6326903.
   PubMed Web of Science ®Google Scholar
• Hill AF, Joiner S, Wadsworth JD, et al. Molecular classification of sporadic Creutzfeldt-Jakob disease. Brain. 2003 Jun;126(Pt 6):1333–1346. PubMed PMID: 12764055.
   PubMed Web of Science ®Google Scholar
• Collinge J, Owen F, Poulter M, et al. Prion dementia without characteristic pathology. Lancet. 1990 Jul 7;336(8706):7–9. PubMed PMID: 1973256.
   PubMed Web of Science ®Google Scholar
• Collinge J, Palmer MS, Sidle KC, et al. Transmission of fatal familial insomnia to laboratory animals. Lancet. 1995 Aug 26;346(8974):569–570. PubMed PMID: 7658786.
   PubMed Web of Science ®Google Scholar
• Salvesen O, Espenes A, Reiten MR, et al. Goats naturally devoid of PrP(C) are resistant to scrapie. Vet Res. 2020 Jan 10;51(1):1. PubMed PMID: 31924264; PubMed Central PMCID: PMCPMC6954626.
   PubMed Web of Science ®Google Scholar
• Bueler H, Aguzzi A, Sailer A, et al. Mice devoid of PrP are resistant to scrapie. Cell. 1993 Jul 2;73(7):1339–1347. PubMed PMID: 8100741.
   PubMed Web of Science ®Google Scholar
• Prusiner SB, Groth D, Serban A, et al. Ablation of the prion protein (PrP) gene in mice prevents scrapie and facilitates production of anti-PrP antibodies. Proc Natl Acad Sci U S A. 1993 Nov 15;90(22):10608–10612. PubMed PMID: 7902565; PubMed Central PMCID: PMCPMC47826.
   PubMed Web of Science ®Google Scholar
• Sakaguchi S, Katamine S, Shigematsu K, et al. Accumulation of proteinase K-resistant prion protein (PrP) is restricted by the expression level of normal PrP in mice inoculated with a mouse-adapted strain of the Creutzfeldt-Jakob disease agent. J Virol. 1995 Dec;69(12):7586–7592. PubMed PMID: 7494265; PubMed Central PMCID: PMCPMC189697.
   PubMed Web of Science ®Google Scholar
• Sollazzo V, Galasso M, Volinia S, et al. Prion proteins (PRNP and PRND) are over-expressed in osteosarcoma. J Orthop Res. 2012 Jun;30(6):1004–1012. PubMed PMID: 22147650.
   PubMed Web of Science ®Google Scholar
• Wiegmans AP, Saunus JM, Ham S. et al. Secreted cellular prion protein binds doxorubicin and correlates with anthracycline resistance in breast cancer. JCI Insight. 2019 Feb 26;5. PubMed PMID: 30830863; PubMed Central PMCID: PMCPMC6483007. DOI:10.1172/jci.insight.124092
   PubMed Web of Science ®Google Scholar
• Lee JH, Yun CW, Lee SH. Cellular prion protein enhances drug resistance of colorectal cancer cells via regulation of a survival signal pathway. Biomol Ther (Seoul). 2018 May 1;26(3):313–321. PubMed PMID: 28822989; PubMed Central PMCID: PMCPMC5933899.
   PubMed Web of Science ®Google Scholar
• Mallucci G, Dickinson A, Linehan J, et al. Depleting neuronal PrP in prion infection prevents disease and reverses spongiosis. Science. 2003 Oct 31;302(5646):871–874. PubMed PMID: 14593181.
   PubMed Web of Science ®Google Scholar
• Bueler H, Fischer M, Lang Y, et al. Normal development and behaviour of mice lacking the neuronal cell-surface PrP protein. Nature. 1992 Apr 16;356(6370):577–582. PubMed PMID: 1373228.
   PubMed Web of Science ®Google Scholar
• Richt JA, Kasinathan P, Hamir AN, et al. Production of cattle lacking prion protein. Nat Biotechnol. 2007 Jan;25(1):132–138. PubMed PMID: 17195841; PubMed Central PMCID: PMCPMC2813193.
   PubMed Web of Science ®Google Scholar
• Benestad SL, Austbo L, Tranulis MA, et al. Healthy goats naturally devoid of prion protein. Vet Res. 2012 Dec;43(1):87. PubMed PMID: 23249298; PubMed Central PMCID: PMCPMC3542104.
   PubMed Web of Science ®Google Scholar
• Collinge J, Whittington MA, Sidle KC, et al. Prion protein is necessary for normal synaptic function. Nature. 1994 Jul 28;370(6487):295–297. PubMed PMID: 8035877.
   PubMed Web of Science ®Google Scholar
• Senatore A, Restelli E, Chiesa R. Synaptic dysfunction in prion diseases: a trafficking problem? Int J Cell Biol. 2013;2013:543803. PubMed PMID: 24369467; PubMed Central PMCID: PMCPMC3863542.
   PubMedGoogle Scholar
• Steinert JR. Prion protein as a mediator of synaptic transmission. Commun Integr Biol. 2015 Jul-Aug;8(4):e1063753. PubMed PMID: 26478992; PubMed Central PMCID: PMCPMC4594542.
   PubMedGoogle Scholar
• Linden R, Martins VR, Prado MA, et al. Physiology of the prion protein. Physiol Rev. 2008 Apr;88(2):673–728. PubMed PMID: 18391177.
   PubMed Web of Science ®Google Scholar
• Malachin G, Reiten MR, Salvesen O, et al. Loss of prion protein induces a primed state of type I interferon-responsive genes. PLoS One. 2017;12(6):e0179881. PubMed PMID: 28651013; PubMed Central PMCID: PMCPMC5484497.
   PubMed Web of Science ®Google Scholar
• Salvesen O, Reiten MR, Kamstra JH, et al. Goats without prion protein display enhanced proinflammatory pulmonary signaling and extracellular matrix remodeling upon systemic lipopolysaccharide challenge. Front Immunol. 2017;8:1722. PubMed PMID: 29270176; PubMed Central PMCID: PMCPMC5723645.
   PubMed Web of Science ®Google Scholar
• Salvesen O, Reiten MR, Espenes A, et al. LPS-induced systemic inflammation reveals an immunomodulatory role for the prion protein at the blood-brain interface. J Neuroinflammation. 2017 May 22;14(1):106. PubMed PMID: 28532450; PubMed Central PMCID: PMCPMC5441080.
   PubMed Web of Science ®Google Scholar
• Wu GR, Mu TC, Gao ZX, et al. Prion protein is required for tumor necrosis factor alpha (TNFalpha)-triggered nuclear factor kappaB (NF-kappaB) signaling and cytokine production. J Biol Chem. 2017 Nov 17;292(46):18747–18759. PubMed PMID: 28900035; PubMed Central PMCID: PMCPMC5704461.
   PubMed Web of Science ®Google Scholar
• White MD, Mallucci GR. Therapy for prion diseases: insights from the use of RNA interference. Prion. 2009 Jul-Sep;3(3):121–128. PubMed PMID: 19597349; PubMed Central PMCID: PMCPMC2802775.
   PubMed Web of Science ®Google Scholar
• Barreca ML, Iraci N, Biggi S, et al. Pharmacological agents targeting the cellular prion protein. Pathogens. 2018 Mar 7;7(1). PubMed PMID: 29518975; PubMed Central PMCID: PMCPMC5874753. DOI:10.3390/pathogens7010027
   PubMed Web of Science ®Google Scholar
• Soto C, Kascsak RJ, Saborio GP, et al. Reversion of prion protein conformational changes by synthetic beta-sheet breaker peptides. Lancet. 2000 Jan 15;355(9199):192–197. PubMed PMID: 10675119.
   PubMed Web of Science ®Google Scholar
• Supattapone S, Wille H, Uyechi L, et al. Branched polyamines cure prion-infected neuroblastoma cells. J Virol. 2001 Apr;75(7):3453–3461. PubMed PMID: 11238871; PubMed Central PMCID: PMCPMC114138.
   PubMed Web of Science ®Google Scholar
• Nilsson KP, Ikenberg K, Aslund A, et al. Structural typing of systemic amyloidoses by luminescent-conjugated polymer spectroscopy. Am J Pathol. 2010 Feb;176(2):563–574. PubMed PMID: 20035056; PubMed Central PMCID: PMCPMC2808065.
   PubMed Web of Science ®Google Scholar
• Nilsson KP, Herland A, Hammarstrom P, et al. Conjugated polyelectrolytes: conformation-sensitive optical probes for detection of amyloid fibril formation. Biochemistry. 2005 Mar 15;44(10):3718–3724. PubMed PMID: 15751948.
   PubMed Web of Science ®Google Scholar
• Margalith I, Suter C, Ballmer B, et al. Polythiophenes inhibit prion propagation by stabilizing prion protein (PrP) aggregates. J Biol Chem. 2012 Jun 1;287(23):18872–18887. PubMed PMID: 22493452; PubMed Central PMCID: PMCPMC3365923.
   PubMed Web of Science ®Google Scholar
• Herrmann US, Schutz AK, Shirani H, et al. Structure-based drug design identifies polythiophenes as antiprion compounds. Sci Transl Med. 2015 Aug 5;7(299):299ra123. PubMed PMID: 26246168.
   PubMed Web of Science ®Google Scholar
• Caspi S, Halimi M, Yanai A, et al. The anti-prion activity of Congo red. Putative mechanism. J Biol Chem. 1998 Feb 6;273(6):3484–3489. PubMed PMID: 9452472.
   PubMed Web of Science ®Google Scholar
• Yakupova EI, Bobyleva LG, Vikhlyantsev IM, et al. Congo Red and amyloids: history and relationship. Biosci Rep. 2019 Jan 31;39(1). PubMed PMID: 30567726; PubMed Central PMCID: PMCPMC6331669. DOI:10.1042/BSR20181415
   PubMed Web of Science ®Google Scholar
• Prusiner SB, McKinley MP, Bowman KA, et al. Scrapie prions aggregate to form amyloid-like birefringent rods. Cell. 1983 Dec;35(2 Pt 1):349–358. PubMed PMID: 6418385.
   PubMed Web of Science ®Google Scholar
• Caughey B, Ernst D, Race RE. Congo red inhibition of scrapie agent replication. J Virol. 1993 Oct;67(10):6270–6272. PubMed PMID: 8103804; PubMed Central PMCID: PMCPMC238051.
   PubMed Web of Science ®Google Scholar
• Ingrosso L, Ladogana A, Pocchiari M. Congo red prolongs the incubation period in scrapie-infected hamsters. J Virol. 1995 Jan;69(1):506–508. PubMed PMID: 7983747; PubMed Central PMCID: PMCPMC188599.
   PubMed Web of Science ®Google Scholar
• Corato M, Ogliari P, Ceciliani F, et al. Doxorubicin and congo red effectiveness on prion infectivity in golden Syrian hamster. Anticancer Res. 2009 Jul;29(7):2507–2512. PubMed PMID: 19596920.
   PubMed Web of Science ®Google Scholar
• Miller-Vedam L, Ghaemmaghami S. Strain specificity and drug resistance in anti-prion therapy. Curr Top Med Chem. 2013;13(19):2397–2406. PubMed PMID: 24059341.
   PubMed Web of Science ®Google Scholar
• Puoti G, Giaccone G, Rossi G, et al. Sporadic Creutzfeldt-Jakob disease: co-occurrence of different types of PrP(Sc) in the same brain. Neurology. 1999 Dec 10;53(9):2173–2176. PubMed PMID: 10599800.
   PubMed Web of Science ®Google Scholar
• Thackray AM, Hopkins L, Lockey R, et al. Emergence of multiple prion strains from single isolates of ovine scrapie. J Gen Virol. 2011 Jun;92(Pt 6):1482–1491. PubMed PMID: 21270287.
   PubMed Web of Science ®Google Scholar
• Mallucci GR, Ratte S, Asante EA, et al. Post-natal knockout of prion protein alters hippocampal CA1 properties, but does not result in neurodegeneration. Embo J. 2002 Feb 1;21(3):202–210. PubMed PMID: 11823413; PubMed Central PMCID: PMCPMC125833.
   PubMed Web of Science ®Google Scholar
• Korth C, May BC, Cohen FE, et al. Acridine and phenothiazine derivatives as pharmacotherapeutics for prion disease. Proc Natl Acad Sci U S A. 2001 Aug 14;98(17):9836–9841. PubMed PMID: 11504948; PubMed Central PMCID: PMCPMC55539.
   PubMed Web of Science ®Google Scholar
• Benito-Leon J. Combined quinacrine and chlorpromazine therapy in fatal familial insomnia. Clin Neuropharmacol. 2004 Jul-Aug;27(4):201–203. PubMed PMID: 15319710.
   PubMed Web of Science ®Google Scholar
• Touil F, Pratt S, Mutter R, et al. Screening a library of potential prion therapeutics against cellular prion proteins and insights into their mode of biological activities by surface plasmon resonance. J Pharm Biomed Anal. 2006 Mar 3;40(4):822–832. PubMed PMID: 16242887.
   PubMed Web of Science ®Google Scholar
• Baral PK, Swayampakula M, Rout MK, et al. Structural basis of prion inhibition by phenothiazine compounds. Structure. 2014 Feb 4;22(2):291–303. PubMed PMID: 24373770.
   PubMed Web of Science ®Google Scholar
• Stincardini C, Massignan T, Biggi S, et al. An antipsychotic drug exerts anti-prion effects by altering the localization of the cellular prion protein. PLoS One. 2017;12(8):e0182589. PubMed PMID: 28787011; PubMed Central PMCID: PMCPMC5546605.
   PubMed Web of Science ®Google Scholar
• Daniel JA, Chau N, Abdel-Hamid MK, et al. Phenothiazine-derived antipsychotic drugs inhibit dynamin and clathrin-mediated endocytosis. Traffic. 2015 Jun;16(6):635–654. PubMed PMID: 25693808.
   PubMed Web of Science ®Google Scholar
• Yamasaki T, Suzuki A, Hasebe R, et al. Comparison of the anti-prion mechanism of four different anti-prion compounds, anti-PrP monoclonal antibody 44B1, pentosan polysulfate, chlorpromazine, and U18666A, in prion-infected mouse neuroblastoma cells. PLoS One. 2014;9(9):e106516. PubMed PMID: 25181483; PubMed Central PMCID: PMCPMC4152300.
   PubMed Web of Science ®Google Scholar
• Massignan T, Cimini S, Stincardini C, et al. A cationic tetrapyrrole inhibits toxic activities of the cellular prion protein. Sci Rep. 2016 Mar;6(1):23180. PubMed PMID: 26976106; PubMed Central PMCID: PMCPMC4791597.
   PubMed Web of Science ®Google Scholar
• Kocisko DA, Caughey WS, Race RE, et al. A porphyrin increases survival time of mice after intracerebral prion infection. Antimicrob Agents Chemother. 2006 Feb;50(2):759–761. PubMed PMID: 16436739; PubMed Central PMCID: PMCPMC1366918.
   PubMed Web of Science ®Google Scholar
• Priola SA, Raines A, Caughey WS. Porphyrin and phthalocyanine antiscrapie compounds. Science. 2000 Feb 25;287(5457):1503–1506. PubMed PMID: 10688802.
   PubMed Web of Science ®Google Scholar
• Rajora MA, Lou JWH, Zheng G. Advancing porphyrin’s biomedical utility via supramolecular chemistry. Chem Soc Rev. 2017 Oct 30;46(21):6433–6469. PubMed PMID: 29048439.
   PubMed Web of Science ®Google Scholar
• Nicoll AJ, Trevitt CR, Tattum MH, et al. Pharmacological chaperone for the structured domain of human prion protein. Proc Natl Acad Sci U S A. 2010 Oct 12;107(41):17610–17615. PubMed PMID: WOS:000282809700032; English.
   PubMed Web of Science ®Google Scholar
• Cavaliere P, Torrent J, Prigent S, et al. Binding of methylene blue to a surface cleft inhibits the oligomerization and fibrillization of prion protein. Biochim Biophys Acta. 2013 Jan;1832(1):20–28. PubMed PMID: 23022479.
   PubMed Web of Science ®Google Scholar
• Yamashita M, Nonaka T, Arai T, et al. Methylene blue and dimebon inhibit aggregation of TDP-43 in cellular models. FEBS Lett. 2009 Jul 21;583(14):2419–2424. PubMed PMID: 19560462.
   PubMed Web of Science ®Google Scholar
• Sontag EM, Lotz GP, Agrawal N, et al. Methylene blue modulates huntingtin aggregation intermediates and is protective in Huntington’s disease models. J Neurosci. 2012 Aug 8;32(32):11109–11119. PubMed PMID: 22875942; PubMed Central PMCID: PMCPMC3546821.
   PubMed Web of Science ®Google Scholar
• Mori T, Koyama N, Segawa T, et al. Methylene blue modulates beta-secretase, reverses cerebral amyloidosis, and improves cognition in transgenic mice. J Biol Chem. 2014 Oct 31;289(44):30303–30317. PubMed PMID: 25157105; PubMed Central PMCID: PMCPMC4215215.
   PubMed Web of Science ®Google Scholar
• Ryou C, Legname G, Peretz D, et al. Differential inhibition of prion propagation by enantiomers of quinacrine. Lab Invest. 2003 Jun;83(6):837–843. PubMed PMID: 12808118.
   PubMed Web of Science ®Google Scholar
• Doh-Ura K, Iwaki T, Caughey B. Lysosomotropic agents and cysteine protease inhibitors inhibit scrapie-associated prion protein accumulation. J Virol. 2000 May;74(10):4894–4897. PubMed PMID: 10775631; PubMed Central PMCID: PMCPMC112015.
   PubMed Web of Science ®Google Scholar
• Vogtherr M, Grimme S, Elshorst B, et al. Antimalarial drug quinacrine binds to C-terminal helix of cellular prion protein. J Med Chem. 2003 Aug 14;46(17):3563–3564. PubMed PMID: 12904059.
   PubMed Web of Science ®Google Scholar
• Kawatake S, Nishimura Y, Sakaguchi S, et al. Surface plasmon resonance analysis for the screening of anti-prion compounds. Biol Pharm Bull. 2006 May;29(5):927–932. PubMed PMID: 16651721.
   PubMed Web of Science ®Google Scholar
• Kamatari YO, Hayano Y, Yamaguchi K, et al. Characterizing antiprion compounds based on their binding properties to prion proteins: implications as medical chaperones. Protein Sci. 2013 Jan;22(1):22–34. PubMed PMID: 23081827; PubMed Central PMCID: PMCPMC3575857.
   PubMed Web of Science ®Google Scholar
• Scoazec JY, Krolak-Salmon P, Casez O, et al. Quinacrine-induced cytolytic hepatitis in sporadic Creutzfeldt-Jakob disease. Ann Neurol. 2003 Apr;53(4):546–547. PubMed PMID: 12666126.
   PubMed Web of Science ®Google Scholar
• Collinge J, Gorham M, Hudson F, et al. Safety and efficacy of quinacrine in human prion disease (PRION-1 study): a patient-preference trial. Lancet Neurol. 2009 Apr;8(4):334–344. PubMed PMID: 19278902; PubMed Central PMCID: PMCPMC2660392.
   PubMed Web of Science ®Google Scholar
• Geschwind MD, Kuo AL, Wong KS, et al. Quinacrine treatment trial for sporadic Creutzfeldt-Jakob disease. Neurology. 2013 Dec 3;81(23):2015–2023. PubMed PMID: 24122181; PubMed Central PMCID: PMCPMC4211922.
   PubMed Web of Science ®Google Scholar
• Ghaemmaghami S, Ahn M, Lessard P, et al. Continuous quinacrine treatment results in the formation of drug-resistant prions. PLoS Pathog. 2009 Nov;5(11):e1000673. PubMed PMID: 19956709; PubMed Central PMCID: PMCPMC2777304.
   PubMed Web of Science ®Google Scholar
• Ahn M, Ghaemmaghami S, Huang Y, et al. Pharmacokinetics of quinacrine efflux from mouse brain via the P-glycoprotein efflux transporter. PLoS One. 2012;7(7):e39112. PubMed PMID: 22768295; PubMed Central PMCID: PMCPMC3388068.
   PubMed Web of Science ®Google Scholar
• Ghaemmaghami S, May BC, Renslo AR, et al. Discovery of 2-aminothiazoles as potent antiprion compounds. J Virol. 2010 Apr;84(7):3408–3412. PubMed PMID: 20032192; PubMed Central PMCID: PMCPMC2838138.
   PubMed Web of Science ®Google Scholar
• Berry DB, Lu D, Geva M, et al. Drug resistance confounding prion therapeutics. Proc Natl Acad Sci U S A. 2013 Oct 29;110(44):E4160–9. PubMed PMID: 24128760; PubMed Central PMCID: PMCPMC3816483.
   PubMed Web of Science ®Google Scholar
• Giles K, Berry DB, Condello C, et al. Different 2-aminothiazole therapeutics produce distinct patterns of scrapie prion neuropathology in mouse brains. J Pharmacol Exp Ther. 2015 Oct;355(1):2–12. PubMed PMID: 26224882; PubMed Central PMCID: PMCPMC4576665.
   PubMed Web of Science ®Google Scholar
• Weissmann C, Aguzzi A. Approaches to therapy of prion diseases. Annu Rev Med. 2005;56:321–344. PubMed PMID: 15660515.
   PubMed Web of Science ®Google Scholar
• Pocchiari M, Schmittinger S, Masullo C. Amphotericin B delays the incubation period of scrapie in intracerebrally inoculated hamsters. J Gen Virol. 1987 Jan;68(Pt 1):219–223. PubMed PMID: 2433387.
   PubMed Web of Science ®Google Scholar
• Adjou KT, Privat N, Demart S, et al. MS-8209, an amphotericin B analogue, delays the appearance of spongiosis, astrogliosis and PrPres accumulation in the brain of scrapie-infected hamsters. J Comp Pathol. 2000 Jan;122(1):3–8. PubMed PMID: 10627386.
   PubMed Web of Science ®Google Scholar
• Masullo C, Macchi G, Xi YG, et al. Failure to ameliorate Creutzfeldt-Jakob disease with amphotericin B therapy. J Infect Dis. 1992 Apr;165(4):784–785. PubMed PMID: 1552216.
   PubMed Web of Science ®Google Scholar
• Gabizon R, Meiner Z, Halimi M, et al. Heparin-like molecules bind differentially to prion-proteins and change their intracellular metabolic fate. J Cell Physiol. 1993 Nov;157(2):319–325. PubMed PMID: 7901226.
   PubMed Web of Science ®Google Scholar
• Warner RG, Hundt C, Weiss S, et al. Identification of the heparan sulfate binding sites in the cellular prion protein. J Biol Chem. 2002 May 24;277(21):18421–18430. PubMed PMID: 11882649.
   PubMed Web of Science ®Google Scholar
• Hundt C, Peyrin JM, Haik S, et al. Identification of interaction domains of the prion protein with its 37-kDa/67-kDa laminin receptor. Embo J. 2001 Nov 1;20(21):5876–5886. PubMed PMID: 11689428; PubMed Central PMCID: PMCPMC125289.
   PubMed Web of Science ®Google Scholar
• Doh-ura K, Ishikawa K, Murakami-Kubo I, et al. Treatment of transmissible spongiform encephalopathy by intraventricular drug infusion in animal models. J Virol. 2004 May;78(10):4999–5006. PubMed PMID: 15113880; PubMed Central PMCID: PMCPMC400350.
   PubMed Web of Science ®Google Scholar
• Bone I, Belton L, Walker AS, et al. Intraventricular pentosan polysulphate in human prion diseases: an observational study in the UK. Eur J Neurol. 2008 May;15(5):458–464. PubMed PMID: 18355301.
   PubMed Web of Science ®Google Scholar
• Parry A, Baker I, Stacey R, et al. Long term survival in a patient with variant Creutzfeldt-Jakob disease treated with intraventricular pentosan polysulphate. J Neurol Neurosurg Psychiatry. 2007 Jul;78(7):733–734. PubMed PMID: 17314188; PubMed Central PMCID: PMCPMC2117700.
   PubMed Web of Science ®Google Scholar
• Terada T, Tsuboi Y, Obi T, et al. Less protease-resistant PrP in a patient with sporadic CJD treated with intraventricular pentosan polysulphate. Acta Neurol Scand. 2010 Feb;121(2):127–130. PubMed PMID: 19804470.
   PubMed Web of Science ®Google Scholar
• Newman PK, Todd NV, Scoones D, et al. Postmortem findings in a case of variant Creutzfeldt-Jakob disease treated with intraventricular pentosan polysulfate. J Neurol Neurosurg Psychiatry. 2014 Aug;85(8):921–924. PubMed PMID: 24554103; PubMed Central PMCID: PMCPMC4112497.
   PubMed Web of Science ®Google Scholar
• Forloni G, Iussich S, Awan T, et al. Tetracyclines affect prion infectivity. Proc Natl Acad Sci U S A. 2002 Aug 6;99(16):10849–10854. PubMed PMID: 12149459; PubMed Central PMCID: PMCPMC125061.
   PubMed Web of Science ®Google Scholar
• De Luigi A, Colombo L, Diomede L, et al. The efficacy of tetracyclines in peripheral and intracerebral prion infection. PLoS One. 2008 Mar 26;3(3):e1888. PubMed PMID: 18365024; PubMed Central PMCID: PMCPMC2268013.
   PubMed Web of Science ®Google Scholar
• Haik S, Marcon G, Mallet A, et al. Doxycycline in Creutzfeldt-Jakob disease: a phase 2, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2014 Feb;13(2):150–158. PubMed PMID: 24411709.
   PubMed Web of Science ®Google Scholar
• Varges D, Manthey H, Heinemann U, et al. Doxycycline in early CJD: a double-blinded randomised phase II and observational study. J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):119–125. PubMed PMID: 27807198; PubMed Central PMCID: PMCPMC5284486.
   PubMed Web of Science ®Google Scholar
• Forloni G, Tettamanti M, Lucca U, et al. Preventive study in subjects at risk of fatal familial insomnia: innovative approach to rare diseases. Prion. 2015;9(2):75–79. PubMed PMID: 25996399; PubMed Central PMCID: PMCPMC4601344.
   PubMed Web of Science ®Google Scholar
• White MD, Farmer M, Mirabile I, et al. Single treatment with RNAi against prion protein rescues early neuronal dysfunction and prolongs survival in mice with prion disease. Proc Natl Acad Sci U S A. 2008 Jul 22;105(29):10238–10243. PubMed PMID: 18632556; PubMed Central PMCID: PMCPMC2474561.
   PubMed Web of Science ®Google Scholar
• Wiedemar N, Hauser DA, Maser P. 100 Years of Suramin. Antimicrob Agents Chemother. 2019 Dec 16;64(3). PubMed PMID: 31844000. DOI:10.1128/AAC.01168-19
   Web of Science ®Google Scholar
• Gilch S, Winklhofer KF, Groschup MH, et al. Intracellular re-routing of prion protein prevents propagation of PrPSc and delays onset of prion disease. Embo J. 2001 Aug 1;20(15):3957–3966. PubMed PMID: 11483499; PubMed Central PMCID: PMCPMC149175.
   PubMed Web of Science ®Google Scholar
• Cooksey C. Hematoxylin and related compounds – an annotated bibliography concerning their origin, properties, chemistry, and certain applications. Biotech Histochem. 2010 Feb;85(1):65–82. PubMed PMID: 19568968.
   PubMed Web of Science ®Google Scholar
• Biggi S, Pancher M, Stincardini C, et al. Identification of compounds inhibiting prion replication and toxicity by removing PrP(C) from the cell surface. J Neurochem. 2020 Jan;152(1):136–150. PubMed PMID: 31264722.
   PubMed Web of Science ®Google Scholar
• Hung M-S, Xu Z, Chen Y, et al. Hematein, a casein kinase II inhibitor, inhibits lung cancer tumor growth in a murine xenograft model. Int J Oncol. 2013 Nov;43(5):1517–1522. PubMed PMID: 24008396; PubMed Central PMCID: PMCPMC3823374.
   PubMed Web of Science ®Google Scholar
• Chen J-M, Gao C, Shi Q, et al. Different expression patterns of CK2 subunits in the brains of experimental animals and patients with transmissible spongiform encephalopathies. Arch Virol. 2008;153(6):1013–1020. PubMed PMID: 18404245.
   PubMed Web of Science ®Google Scholar
• Zamponi E, Buratti F, Cataldi G, et al. Prion protein inhibits fast axonal transport through a mechanism involving casein kinase 2. PLoS One. 2017;12(12):e0188340. PubMed PMID: 29261664; PubMed Central PMCID: PMCPMC5737884.
   PubMed Web of Science ®Google Scholar
• Heisler FF, Pechmann Y, Wieser I, et al. Muskelin coordinates PrPC lysosome versus exosome targeting and impacts prion disease progression. Neuron. 2018 Sep 19;99(6):1155–1169 e9. PubMed PMID: 30174115.
   PubMed Web of Science ®Google Scholar
• Lee EC, Kim SH, Park SJ. Safety and efficacy of once-daily prolonged-release tacrolimus in living donor liver transplantation: an open-label, prospective, single-arm, phase 4 study. Ann Transplant. 2018 Oct 12;23:713–720. PubMed PMID: 30310047; PubMed Central PMCID: PMCPMC6248274.
   PubMed Web of Science ®Google Scholar
• Nakagaki T, Satoh K, Ishibashi D, et al. FK506 reduces abnormal prion protein through the activation of autolysosomal degradation and prolongs survival in prion-infected mice. Autophagy. 2013 Sep;9(9):1386–1394. PubMed PMID: 23800841.
   PubMed Web of Science ®Google Scholar
• Phadwal K, Kurian D, Salamat MKF, et al. Spermine increases acetylation of tubulins and facilitates autophagic degradation of prion aggregates. Sci Rep. 2018 Jul 3;8(1):10004. PubMed PMID: 29968775; PubMed Central PMCID: PMCPMC6030104.
   PubMed Web of Science ®Google Scholar
• Rogaeva E, Meng Y, Lee JH, et al. The neuronal sortilin-related receptor SORL1 is genetically associated with Alzheimer disease. Nat Genet. 2007 Feb;39(2):168–177. PubMed PMID: 17220890; PubMed Central PMCID: PMCPMC2657343.
   PubMed Web of Science ®Google Scholar
• Nykjaer A, Willnow TE. Sortilin: a receptor to regulate neuronal viability and function. Trends Neurosci. 2012 Apr;35(4):261–270. PubMed PMID: 22341525.
   PubMed Web of Science ®Google Scholar
• Uchiyama K, Tomita M, Yano M, et al. Prions amplify through degradation of the VPS10P sorting receptor sortilin. PLoS Pathog. 2017 Jun;13(6):e1006470. PubMed PMID: 28665987; PubMed Central PMCID: PMCPMC5509376.
   PubMed Web of Science ®Google Scholar
• Sakaguchi S, Uchiyama K. Novel amplification mechanism of prions through disrupting sortilin-mediated trafficking. Prion. 2017 Nov 2;11(6):398–404. PubMed PMID: 29099278; PubMed Central PMCID: PMCPMC5786358.
   PubMed Web of Science ®Google Scholar
• Heppner FL, Musahl C, Arrighi I, et al. Prevention of scrapie pathogenesis by transgenic expression of anti-prion protein antibodies. Science. 2001 Oct 5;294(5540):178–182. PubMed PMID: 11546838.
   PubMed Web of Science ®Google Scholar
• White AR, Enever P, Tayebi M, et al. Monoclonal antibodies inhibit prion replication and delay the development of prion disease. Nature. 2003 Mar 6;422(6927):80–83. PubMed PMID: 12621436.
   PubMed Web of Science ®Google Scholar
• Barry RA, Prusiner SB. Monoclonal antibodies to the cellular and scrapie prion proteins. J Infect Dis. 1986 Sep;154(3):518–521. PubMed PMID: 3090160.
   PubMed Web of Science ®Google Scholar
• Polymenidou M, Heppner FL, Pellicioli EC, et al. Humoral immune response to native eukaryotic prion protein correlates with anti-prion protection. Proc Natl Acad Sci U S A. 2004 Oct 5;101(Suppl 2):14670–14676. PubMed PMID: 15292505; PubMed Central PMCID: PMCPMC521983.
   PubMed Web of Science ®Google Scholar
• Polymenidou M, Moos R, Scott M, et al. The POM monoclonals: a comprehensive set of antibodies to non-overlapping prion protein epitopes. PLoS One. 2008;3(12):e3872. PubMed PMID: 19060956; PubMed Central PMCID: PMCPMC2592702.
   PubMed Web of Science ®Google Scholar
• Schwarz A, Kratke O, Burwinkel M, et al. Immunisation with a synthetic prion protein-derived peptide prolongs survival times of mice orally exposed to the scrapie agent. Neurosci Lett. 2003 Oct 30;350(3):187–189. PubMed PMID: 14550926.
   PubMed Web of Science ®Google Scholar
• Solforosi L, Criado JR, McGavern DB, et al. Cross-linking cellular prion protein triggers neuronal apoptosis in vivo. Science. 2004 Mar 5;303(5663):1514–1516. PubMed PMID: 14752167.
   PubMed Web of Science ®Google Scholar
• Klohn PC, Farmer M, Linehan JM, et al. PrP antibodies do not trigger mouse hippocampal neuron apoptosis. Science. 2012 Jan 6;335(6064):52. PubMed PMID: 22223800.
   PubMed Web of Science ®Google Scholar
• Dyer C. British man with CJD gets experimental treatment in world first. BMJ. 2018 Oct 31;363:k4608. PubMed PMID: 30381386.
   PubMed Web of Science ®Google Scholar
• Sonati T, Reimann RR, Falsig J, et al. The toxicity of antiprion antibodies is mediated by the flexible tail of the prion protein. Nature. 2013 Sep 5;501(7465):102–106. PubMed PMID: 23903654.
   PubMed Web of Science ®Google Scholar
• Reimann RR, Sonati T, Hornemann S, et al. Differential toxicity of antibodies to the prion protein. PLoS Pathog. 2016 Jan;12(1):e1005401. PubMed PMID: 26821311; PubMed Central PMCID: PMCPMC4731068.
   PubMed Web of Science ®Google Scholar
• Wu B, McDonald AJ, Markham K. et al. The N-terminus of the prion protein is a toxic effector regulated by the C-terminus. Elife. 2017 May 20;6. PubMed PMID: 28527237; PubMed Central PMCID: PMCPMC5469617. DOI:10.7554/eLife.23473
   Web of Science ®Google Scholar
• Pardridge WM. Blood-brain barrier and delivery of protein and gene therapeutics to brain. Front Aging Neurosci. 2019;11:373. PubMed PMID: 31998120; PubMed Central PMCID: PMCPMC6966240.
   PubMed Web of Science ®Google Scholar
• Nikles D, Bach P, Boller K, et al. Circumventing tolerance to the prion protein (PrP): vaccination with PrP-displaying retrovirus particles induces humoral immune responses against the native form of cellular PrP. J Virol. 2005 Apr;79(7):4033–4042. PubMed PMID: 15767405; PubMed Central PMCID: PMCPMC1061525.
   PubMed Web of Science ®Google Scholar
• Handisurya A, Gilch S, Winter D, et al. Vaccination with prion peptide-displaying papillomavirus-like particles induces autoantibodies to normal prion protein that interfere with pathologic prion protein production in infected cells. Febs J. 2007 Apr;274(7):1747–1758. PubMed PMID: 17313482; PubMed Central PMCID: PMCPMC3792370.
   PubMed Web of Science ®Google Scholar
• Huston JS, Levinson D, Mudgett-Hunter M, et al. Protein engineering of antibody binding sites: recovery of specific activity in an anti-digoxin single-chain Fv analogue produced in Escherichia coli. Proc Natl Acad Sci U S A. 1988 Aug;85(16):5879–5883. PubMed PMID: 3045807; PubMed Central PMCID: PMCPMC281868.
   PubMed Web of Science ®Google Scholar
• Donofrio G, Heppner FL, Polymenidou M, et al. Paracrine inhibition of prion propagation by anti-PrP single-chain Fv miniantibodies. J Virol. 2005 Jul;79(13):8330–8338. PubMed PMID: 15956578; PubMed Central PMCID: PMCPMC1143714.
   PubMed Web of Science ®Google Scholar
• Wuertzer CA, Sullivan MA, Qiu X, et al. CNS delivery of vectored prion-specific single-chain antibodies delays disease onset. Mol Ther. 2008 Mar;16(3):481–486. PubMed PMID: 18180775.
   PubMed Web of Science ®Google Scholar
• Moda F, Vimercati C, Campagnani I, et al. Brain delivery of AAV9 expressing an anti-PrP monovalent antibody delays prion disease in mice. Prion. 2012 Sep-Oct;6(4):383–390. PubMed PMID: 22842862; PubMed Central PMCID: PMCPMC3609068.
   PubMed Web of Science ®Google Scholar
• Cardinale A, Biocca S. Gene-based antibody strategies for prion diseases. Int J Cell Biol. 2013;2013:710406. PubMed PMID: 24027584; PubMed Central PMCID: PMCPMC3763265.
   PubMedGoogle Scholar
• Cardinale A, Filesi I, Vetrugno V, et al. Trapping prion protein in the endoplasmic reticulum impairs PrPC maturation and prevents PrPSc accumulation. J Biol Chem. 2005 Jan 7;280(1):685–694. PubMed PMID: 15513919.
   PubMed Web of Science ®Google Scholar
• Filesi I, Cardinale A, Mattei S, et al. Selective re-routing of prion protein to proteasomes and alteration of its vesicular secretion prevent PrP(Sc) formation. J Neurochem. 2007 Jun;101(6):1516–1526. PubMed PMID: 17542810.
   PubMed Web of Science ®Google Scholar
• Jones M, Wight D, McLoughlin V, et al. An antibody to the aggregated synthetic prion protein peptide (PrP106-126) selectively recognizes disease-associated prion protein (PrP) from human brain specimens. Brain Pathol. 2009 Apr;19(2):293–302. PubMed PMID: 18507665.
   PubMed Web of Science ®Google Scholar
• Tayebi M, Jones DR, Taylor WA, et al. PrP(Sc)-specific antibodies with the ability to immunodetect prion oligomers. PLoS One. 2011;6(5):e19998. PubMed PMID: 21625515; PubMed Central PMCID: PMCPMC3098279.
   PubMed Web of Science ®Google Scholar
• Saijo E, Hughson AG, Raymond GJ, et al. PrPSc-specific antibody reveals C-terminal conformational differences between prion strains. J Virol. 2016 May 15;90(10):4905–4913. PubMed PMID: 26937029; PubMed Central PMCID: PMCPMC4859706.
   PubMed Web of Science ®Google Scholar
• Song CH, Furuoka H, Kim CL, et al. Effect of intraventricular infusion of anti-prion protein monoclonal antibodies on disease progression in prion-infected mice. J Gen Virol. 2008 Jun;89(Pt 6):1533–1544. PubMed PMID: 18474571.
   PubMed Web of Science ®Google Scholar
• Senatore A, Frontzek K, Emmenegger M, et al. Protective anti-prion antibodies in human immunoglobulin repertoires. BioRxiv 2020. 2020.02.05.933721. DOI:10.1101/2020.02.05.933721v1.full.
   Google Scholar
• Williamson RA, Peretz D, Smorodinsky N, et al. Circumventing tolerance to generate autologous monoclonal antibodies to the prion protein. Proc Natl Acad Sci USA. 1996 Jul 9;93(14):7279–7282. PubMed PMID: 8692983; PubMed Central PMCID: PMCPMC38974.
   PubMed Web of Science ®Google Scholar
• Williamson RA, Peretz D, Pinilla C, et al. Mapping the prion protein using recombinant antibodies. J Virol. 1998 Nov;72(11):9413–9418. PubMed PMID: 9765500; PubMed Central PMCID: PMCPMC110372.
   PubMed Web of Science ®Google Scholar
• Moroncini G, Kanu N, Solforosi L, et al. Motif-grafted antibodies containing the replicative interface of cellular PrP are specific for PrPSc. Proc Natl Acad Sci U S A. 2004 Jul 13;101(28):10404–10409. PubMed PMID: 15240877; PubMed Central PMCID: PMCPMC478584.
   PubMed Web of Science ®Google Scholar
• Gilch S, Schatzl HM. Aptamers against prion proteins and prions. Cell Mol Life Sci. 2009 Aug;66(15):2445–2455. PubMed PMID: 19396399.
   PubMed Web of Science ®Google Scholar
• Proske D, Gilch S, Wopfner F, et al. Prion-protein-specific aptamer reduces PrPSc formation. Chembiochem. 2002 Aug 2;3(8):717–725. PubMed PMID: 12203970.
   PubMed Web of Science ®Google Scholar
• Gilch S, Kehler C, Schatzl HM. Peptide aptamers expressed in the secretory pathway interfere with cellular PrPSc formation. J Mol Biol. 2007 Aug 10;371(2):362–373. PubMed PMID: 17574575.
   PubMed Web of Science ®Google Scholar
• Weissmann C, Bueler H, Fischer M, et al. PrP-deficient mice are resistant to scrapie. Ann N Y Acad Sci. 1994 Jun;6(724):235–240. PubMed PMID: 8030944.
   PubMed Web of Science ®Google Scholar
• Sailer A, Bueler H, Fischer M, et al. No propagation of prions in mice devoid of PrP. Cell. 1994 Jul 1;77(7):967–968. PubMed PMID: 7912659.
   PubMed Web of Science ®Google Scholar
• Bueler H, Raeber A, Sailer A, et al. High prion and PrPSc levels but delayed onset of disease in scrapie-inoculated mice heterozygous for a disrupted PrP gene. Mol Med. 1994 Nov;1(1):19–30. PubMed PMID: 8790598; PubMed Central PMCID: PMCPMC2229922.
   PubMed Web of Science ®Google Scholar
• Gambetti P, Dong Z, Yuan J, et al. A novel human disease with abnormal prion protein sensitive to protease. Ann Neurol. 2008 Jun;63(6):697–708. PubMed PMID: 18571782; PubMed Central PMCID: PMCPMC2767200.
   PubMed Web of Science ®Google Scholar
• Collinge J, Palmer MS, Sidle KC, et al. Unaltered susceptibility to BSE in transgenic mice expressing human prion protein. Nature. 1995 Dec 21- 28;378(6559):779–783. PubMed PMID: 8524411.
   PubMed Web of Science ®Google Scholar
• Colling SB, Collinge J, Jefferys JG. Hippocampal slices from prion protein null mice: disrupted Ca(2+)-activated K+ currents. Neurosci Lett. 1996 May 3;209(1):49–52. PubMed PMID: 8734907.
   PubMed Web of Science ®Google Scholar
• Pfeifer A, Eigenbrod S, Al-Khadra S, et al. Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice. J Clin Invest. 2006 Dec;116(12):3204–3210. PubMed PMID: 17143329; PubMed Central PMCID: PMCPMC1679709.
   PubMed Web of Science ®Google Scholar
• Pulford B, Reim N, Bell A, et al. Liposome-siRNA-peptide complexes cross the blood-brain barrier and significantly decrease PrP on neuronal cells and PrP in infected cell cultures. PLoS One. 2010 Jun 14;5(6):e11085. PubMed PMID: 20559428; PubMed Central PMCID: PMCPMC2885418.
   PubMed Web of Science ®Google Scholar
• Manson JC, Clarke AR, McBride PA, et al. PrP gene dosage determines the timing but not the final intensity or distribution of lesions in scrapie pathology. Neurodegeneration. 1994 Dec;3(4):331–340. PubMed PMID: 7842304.
   PubMedGoogle Scholar
• Minikel EV, Vallabh SM, Lek M, et al. Quantifying prion disease penetrance using large population control cohorts. Sci Transl Med. 2016 Jan 20;8(322):322ra9. PubMed PMID: 26791950; PubMed Central PMCID: PMCPMC4774245.
   PubMed Web of Science ®Google Scholar
• Tremblay P, Meiner Z, Galou M, et al. Doxycycline control of prion protein transgene expression modulates prion disease in mice. Proc Natl Acad Sci U S A. 1998 Oct 13;95(21):12580–12585. PubMed PMID: 9770528; PubMed Central PMCID: PMCPMC22873.
   PubMed Web of Science ®Google Scholar
• Safar JG, DeArmond SJ, Kociuba K, et al. Prion clearance in bigenic mice. J Gen Virol. 2005 Oct;86(Pt 10):2913–2923. PubMed PMID: 16186247.
   PubMed Web of Science ®Google Scholar
• Mallucci GR, White MD, Farmer M, et al. Targeting cellular prion protein reverses early cognitive deficits and neurophysiological dysfunction in prion-infected mice. Neuron. 2007 Feb 1;53(3):325–335. PubMed PMID: 17270731.
   PubMed Web of Science ®Google Scholar
• Mirabile I, Jat PS, Brandner S, et al. Identification of clinical target areas in the brainstem of prion-infected mice. Neuropathol Appl Neurobiol. 2015 Aug;41(5):613–630. PubMed PMID: 25311251; PubMed Central PMCID: PMCPMC4949711.
   PubMed Web of Science ®Google Scholar
• Raeber AJ, Race RE, Brandner S, et al. Astrocyte-specific expression of hamster prion protein (PrP) renders PrP knockout mice susceptible to hamster scrapie. Embo J. 1997 Oct 15;16(20):6057–6065. PubMed PMID: 9321385; PubMed Central PMCID: PMCPMC1326289.
   PubMed Web of Science ®Google Scholar
• Krejciova Z, Alibhai J, Zhao C, et al. Human stem cell-derived astrocytes replicate human prions in a PRNP genotype-dependent manner. J Exp Med. 2017 Dec 4;214(12):3481–3495. PubMed PMID: 29141869; PubMed Central PMCID: PMCPMC5716027.
   PubMed Web of Science ®Google Scholar
• Smith HL, Freeman OJ, Butcher AJ, et al. Astrocyte unfolded protein response induces a specific reactivity state that causes non-cell-autonomous neuronal degeneration. Neuron. 2020 Mar 4;105(5):855–866 e5. PubMed PMID: 31924446; PubMed Central PMCID: PMCPMC7054837.
   PubMed Web of Science ®Google Scholar
• Houston F, Goldmann W, Foster J, et al. Comparative susceptibility of sheep of different origins, breeds and PRNP genotypes to challenge with bovine spongiform encephalopathy and scrapie. PLoS One. 2015;10(11):e0143251. PubMed PMID: 26587837; PubMed Central PMCID: PMCPMC4654545.
   PubMed Web of Science ®Google Scholar
• Espinosa JC, Marin-Moreno A, Aguilar-Calvo P. et al. Porcine prion protein as a paradigm of limited susceptibility to prion strain propagation. J Infect Dis. 2020 Jan 9; PubMed PMID: 31919511. DOI:10.1093/infdis/jiz646.
   PubMed Web of Science ®Google Scholar
• Vidal E, Fernandez-Borges N, Erana H, et al. Dogs are resistant to prion infection, due to the presence of aspartic or glutamic acid at position 163 of their prion protein. Faseb J. 2020 Mar;34(3):3969–3982. PubMed PMID: 31944411.
   PubMed Web of Science ®Google Scholar
• Bourkas MEC, Arshad H, Al-Azzawi ZAM, et al. Engineering a murine cell line for the stable propagation of hamster prions. J Biol Chem. 2019 Mar 29;294(13):4911–4923. PubMed PMID: 30705093; PubMed Central PMCID: PMCPMC6442044.
   PubMed Web of Science ®Google Scholar
• Walia R, Ho CC, Lee C, et al. Gene-edited murine cell lines for propagation of chronic wasting disease prions. Sci Rep. 2019 Aug 1;9(1):11151. PubMed PMID: 31371793; PubMed Central PMCID: PMCPMC6673760.
   PubMed Web of Science ®Google Scholar
• Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998 Feb 19;391(6669):806–811. PubMed PMID: 9486653.
   PubMed Web of Science ®Google Scholar
• Montgomery MK, Xu S, Fire A. RNA as a target of double-stranded RNA-mediated genetic interference in Caenorhabditis elegans. Proc Natl Acad Sci U S A. 1998 Dec 22;95(26):15502–15507. PubMed PMID: 9860998; PubMed Central PMCID: PMCPMC28072.
   PubMed Web of Science ®Google Scholar
• Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science. 2002 Apr 19;296(5567):550–553. PubMed PMID: 11910072.
   PubMed Web of Science ®Google Scholar
• Schlachetzki F, Zhang Y, Boado RJ, et al. Gene therapy of the brain: the trans-vascular approach. Neurology. 2004 Apr 27;62(8):1275–1281. PubMed PMID: 15111662.
   PubMed Web of Science ®Google Scholar
• Shiekh FA. Blood-brain barrier: a real obstacle for therapeutics. Int J Nanomedicine. 2012;7:4065–4066. author reply 4067. PubMed PMID: 22888242; PubMed Central PMCID: PMCPMC3414082.
   PubMed Web of Science ®Google Scholar
• Kumar P, Wu H, McBride JL, et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature. 2007 Jul 5;448(7149):39–43. PubMed PMID: 17572664.
   PubMed Web of Science ®Google Scholar
• Lehmann S, Relano-Gines A, Resina S, et al. Systemic delivery of siRNA down regulates brain prion protein and ameliorates neuropathology in prion disorder. PLoS One. 2014;9(2):e88797. PubMed PMID: 24551164; PubMed Central PMCID: PMCPMC3925167.
   PubMed Web of Science ®Google Scholar
• Bender H, Noyes N, Annis JL, et al. PrPC knockdown by liposome-siRNA-peptide complexes (LSPCs) prolongs survival and normal behavior of prion-infected mice immunotolerant to treatment. PLoS One. 2019;14(7):e0219995. PubMed PMID: 31329627; PubMed Central PMCID: PMCPMC6645518.
   PubMed Web of Science ®Google Scholar
• Ahn M, Bajsarowicz K, Oehler A, et al. Convection-enhanced delivery of AAV2-PrPshRNA in prion-infected mice. PLoS One. 2014;9(5):e98496. PubMed PMID: 24866748; PubMed Central PMCID: PMCPMC4035323.
   PubMed Web of Science ®Google Scholar
• Leavitt BR, Tabrizi SJ. Antisense oligonucleotides for neurodegeneration. Science. 2020 Mar 27;367(6485):1428–1429. PubMed PMID: 32217715.
   PubMed Web of Science ®Google Scholar
• Schoch KM, Miller TM. Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases. Neuron. 2017 Jun 21;94(6):1056–1070. PubMed PMID: 28641106; PubMed Central PMCID: PMCPMC5821515.
   PubMed Web of Science ®Google Scholar
• Smith RA, Miller TM, Yamanaka K, et al. Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest. 2006 Aug;116(8):2290–2296. PubMed PMID: 16878173; PubMed Central PMCID: PMCPMC1518790.
   PubMed Web of Science ®Google Scholar
• Kordasiewicz HB, Stanek LM, Wancewicz EV, et al. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron. 2012 Jun 21;74(6):1031–1044. PubMed PMID: 22726834; PubMed Central PMCID: PMCPMC3383626.
   PubMed Web of Science ®Google Scholar
• Southwell AL, Skotte NH, Bennett CF, et al. Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases. Trends Mol Med. 2012 Nov;18(11):634–643. PubMed PMID: 23026741.
   PubMed Web of Science ®Google Scholar
• Southwell AL, Kordasiewicz HB, Langbehn D, et al. Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease. Sci Transl Med. 2018 Oct 3;10(461). PubMed PMID: 30282695. DOI:10.1126/scitranslmed.aar3959
   PubMed Web of Science ®Google Scholar
• Scoles DR, Meera P, Schneider MD, et al. Antisense oligonucleotide therapy for spinocerebellar ataxia type 2. Nature. 2017 Apr 20;544(7650):362–366. PubMed PMID: 28405024; PubMed Central PMCID: PMCPMC6625650.
   PubMed Web of Science ®Google Scholar
• Scoles DR, Pulst SM. Oligonucleotide therapeutics in neurodegenerative diseases. RNA Biol. 2018;15(6):707–714. PubMed PMID: 29560813; PubMed Central PMCID: PMCPMC6152438.
   PubMed Web of Science ®Google Scholar
• Scoles DR, Minikel EV, Pulst SM. Antisense oligonucleotides: A primer. Neurol Genet. 2019 Apr;5(2):e323. PubMed PMID: 31119194; PubMed Central PMCID: PMCPMC6501637.
   PubMed Web of Science ®Google Scholar
• Wurster CD, Ludolph AC. Antisense oligonucleotides in neurological disorders. Ther Adv Neurol Disord. 2018;11:1756286418776932. PubMed PMID: 29854003; PubMed Central PMCID: PMCPMC5971383.
   Web of Science ®Google Scholar
• DeVos SL, Miller RL, Schoch KM, et al. Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy. Sci Transl Med. 2017 Jan 25;9(374). PubMed PMID: 28123067; PubMed Central PMCID: PMCPMC5792300. DOI:10.1126/scitranslmed.aag0481
   PubMed Web of Science ®Google Scholar
• Singh NK, Singh NN, Androphy EJ, et al. Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron. Mol Cell Biol. 2006 Feb;26(4):1333–1346. PubMed PMID: 16449646; PubMed Central PMCID: PMCPMC1367187.
   PubMed Web of Science ®Google Scholar
• Schoch KM, DeVos SL, Miller RL, et al. Increased 4R-tau induces pathological changes in a human-tau mouse model. Neuron. 2016 Jun 1;90(5):941–947. PubMed PMID: 27210553; PubMed Central PMCID: PMCPMC5040069.
   PubMed Web of Science ®Google Scholar
• Bennett CF, Baker BF, Pham N, et al. Pharmacology of antisense drugs. Annu Rev Pharmacol Toxicol. 2017 Jan;6(57):81–105. PubMed PMID: 27732800.
   PubMed Web of Science ®Google Scholar
• Stein CA, Subasinghe C, Shinozuka K, et al. Physicochemical properties of phosphorothioate oligodeoxynucleotides. Nucleic Acids Res. 1988 Apr 25;16(8):3209–3221. PubMed PMID: 2836790; PubMed Central PMCID: PMCPMC336489.
   PubMed Web of Science ®Google Scholar
• Goel S, Desai K, Macapinlac M, et al. A phase I safety and dose escalation trial of docetaxel combined with GEM231, a second generation antisense oligonucleotide targeting protein kinase A R1alpha in patients with advanced solid cancers. Invest New Drugs. 2006 Mar;24(2):125–134. PubMed PMID: 16683205.
   PubMed Web of Science ®Google Scholar
• Janssen HL, Reesink HW, Lawitz EJ, et al. Treatment of HCV infection by targeting microRNA. N Engl J Med. 2013 May 2;368(18):1685–1694. PubMed PMID: 23534542.
   PubMed Web of Science ®Google Scholar
• Santos RD, Duell PB, East C, et al. Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension. Eur Heart J. 2015 Mar 1;36(9):566–575. PubMed PMID: 24366918; PubMed Central PMCID: PMCPMC4344956.
   PubMed Web of Science ®Google Scholar
• Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, et al. Targeting huntingtin expression in patients with Huntington’s disease. N Engl J Med. 2019 Jun 13;380(24):2307–2316. PubMed PMID: 31059641.
   PubMed Web of Science ®Google Scholar
• Rigo F, Chun SJ, Norris DA, et al. Pharmacology of a central nervous system delivered 2ʹ-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. J Pharmacol Exp Ther. 2014 Jul;350(1):46–55. PubMed PMID: 24784568; PubMed Central PMCID: PMCPMC4056267.
   PubMed Web of Science ®Google Scholar
• Miller TM, Pestronk A, David W, et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013 May;12(5):435–442. PubMed PMID: 23541756; PubMed Central PMCID: PMCPMC3712285.
   PubMed Web of Science ®Google Scholar
• Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy. Neurology. 2016 Mar 8;86(10):890–897. PubMed PMID: 26865511; PubMed Central PMCID: PMCPMC4782111.
   PubMed Web of Science ®Google Scholar
• Kocisko DA, Vaillant A, Lee KS, et al. Potent antiscrapie activities of degenerate phosphorothioate oligonucleotides. Antimicrob Agents Chemother. 2006 Mar;50(3):1034–1044. PubMed PMID: 16495266; PubMed Central PMCID: PMCPMC1426446.
   PubMed Web of Science ®Google Scholar
• Nazor Friberg K, Hung G, Wancewicz E, et al. Intracerebral infusion of antisense oligonucleotides into prion-infected mice. Mol Ther Nucleic Acids. 2012 Feb;7(1):e9. PubMed PMID: 23344724; PubMed Central PMCID: PMCPMC3381600.
   PubMedGoogle Scholar
• Karpuj MV, Giles K, Gelibter-Niv S, et al. Phosphorothioate oligonucleotides reduce PrP levels and prion infectivity in cultured cells. Mol Med. 2007 Mar-Apr;13(3–4):190–198. PubMed PMID: 17592554; PubMed Central PMCID: PMCPMC1892763.
   PubMed Web of Science ®Google Scholar
• Raymond GJ, Zhao HT, Race B. et al. Antisense oligonucleotides extend survival of prion-infected mice. JCI Insight. 2019 Jul 30;5. PubMed PMID: 31361599; PubMed Central PMCID: PMCPMC6777807. DOI:10.1172/jci.insight.131175
   PubMed Web of Science ®Google Scholar
• Minikel EV, Zhao HT, Le J, et al. Prion protein lowering is a disease-modifying therapy across prion strains, disease stages, and endpoints. BioRxiv 2020. 2020.03.27.011940. DOI:10.1101/2020.03.27.011940v2.abstract.
   Google Scholar