Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment

References

• Benson MD, Kincaid JC. The molecular biology and clinical features of amyloid neuropathy. Muscle Nerve 2007;36:411–423.
   PubMed Web of Science ®Google Scholar
• Benson MD. Amyloidosis and other protein deposition diseases. In: Rimoin DL, Connor JM, Pyeritz RE, Korf BR, editors. Emery and Rimoin's principles and practice of medical genetics. 5th ed., Vol. 2, Chapter 83. Churchill Livingstone publishers; 2005. pp. 1821–1834.
   Google Scholar
• Martone RL, Herbert J, Dwork A, Schon EA. Transthyretin is synthesized in the mammalian eye. Biochem Biophys Res Commun 1988;151:905–912.
   PubMed Web of Science ®Google Scholar
• Brett M, Persey MR, Reilly MM, Revesz T, Booth DR, Booth SE, Hawkins PN, Pepys MB, Morgan-Hughes JA. Transthyretin Leu12Pro is associated with systemic, neuropathic and leptomeningeal amyloidosis. Brain 1999;122:183–190.
   PubMed Web of Science ®Google Scholar
• Garzuly F, Vidal R, Wisniewski T, Brittig F, Budka H. Familial meningocerebro-vascular amyloidosis, Hungarian type, with mutant transthyretin (TTR Asp18Gly). Neurology 1996;47:1562–1567.
   PubMed Web of Science ®Google Scholar
• Vidal R, Garzuly F, Budka H, Lalowski M, Linke RP, Brittig F, Frangione B, Wisniewski T. Meningocerebrovascular amyloidosis associated with a novel transthyretin mis-sense mutation at codon 18 (TTR D18G). Am J Pathol 1996;148:361–366.
   PubMedGoogle Scholar
• Sekijima Y, Hammarström P, Matsumura M, Shimizu Y, Iwata M, Tokuda T, Ikeda S, Kelly JW. Energetic characteristics of the new transthyretin variant A25T may explain its atypical central nervous system pathology. Lab Invest 2003;83:409–417.
   PubMed Web of Science ®Google Scholar
• Shimizu Y, Takeuchi M, Matsumura M, Tokuda T, Iwata M. A case of biopsy-proven leptomeningeal amyloidosis and intravenous Ig-responsive polyneuropathy associated with the Ala25Thr transthyretin gene mutation. Amyloid 2006;13:37–41.
   PubMed Web of Science ®Google Scholar
• Herrick MK, DeBruyne K, Horoupian DS, Skare J, Vanefsky MA, Ong T. Massive leptomeningeal amyloidosis associated with a Val30Met transthyretin gene. Neurology 1996;47:988–992.
   PubMed Web of Science ®Google Scholar
• Goren H, Steinberg MC, Farboody GH. Familial oculoleptomeningeal amyloidosis. Brain 1980;103:473–495.
   PubMed Web of Science ®Google Scholar
• Petersen RB, Goren H, Cohen M, Richardson SL, Tresser N, Lynn A, Gali M, Estes M, Gambetti P. Transthyretin amyloidosis: a new mutation associated with dementia. Ann Neurol 1997;41:307–313.
   PubMed Web of Science ®Google Scholar
• Mascalchi M, Salvi F, Pirini MG, D'Errico A, Ferlini A, Lolli F, Plasmati R, Tessa C, Villari N, Tassinari CA. Transthyretin amyloidosis and superficial siderosis of the CNS. Neurology 1999;53:1498–1503.
   PubMed Web of Science ®Google Scholar
• Nakagawa K, Sheikh SI, Snuderl M, Frosch MP, Greenberg SM. A new Thr49Pro transthyretin gene mutation associated with leptomeningeal amyloidosis. J Neurol Sci 2008;272:186–190.
   PubMedGoogle Scholar
• Ellie E, Camou F, Vital A, Rummens C, Grateau G, Delpech M, Valleix S. Recurrent subarachnoid hemorrhage associated with a new transthyretin variant (Gly53Glu). Neurology 2001;57:135–137.
   PubMed Web of Science ®Google Scholar
• Uemichi T, Uitti RJ, Koeppen AH, Donat JR, Benson MD. Oculoleptomeningeal amyloidosis associated with a new transthyretin variant Ser64. Arch Neurol 1999;56:1152–1155.
   PubMedGoogle Scholar
• Blevins G, Macaulay R, Harder S, Fladeland D, Yamashita T, Yazaki M, Hamidi Asl K, Benson MD, Donat JR. Oculoleptomeningeal amyloidosis in a large kindred with a new transthyretin variant Tyr69His. Neurology 2003;60:1625–1630.
   PubMed Web of Science ®Google Scholar
• Ueno S, Uemichi T, Yorifuji S, Tarui S. A novel variant of transthyretin (Tyr114 to Cys) deduced from the nucleotide sequences of gene fragments from familial amyloidotic polyneuropathy in Japanese sibling cases. Biochem Biophys Res Commun 1990;169:143–147.
   PubMed Web of Science ®Google Scholar
• Southwell BR, Duan W, Alcorn D, Brack C, Richardson SJ, Köhrle J, Schreiber G. Thyroxine transport to the brain: role of protein synthesis by the choroid plexus. Endocrinology 1993;133:2116–2126.
   PubMed Web of Science ®Google Scholar
• Motojima K, Goto S. Brain-specific expression of transthyretin mRNA as revealed by cDNA cloning from brain. FEBS Lett 1989;258:103–105.
   PubMedGoogle Scholar
• Mitsuhashi S, Yazaki M, Tokuda T, Sekijima Y, Washimi Y, Shimizu Y, Ando Y, Benson MD, Ikeda S. Biochemical characteristics of variant transthyretins causing hereditary leptomeningeal amyloidosis. Amyloid 2005;12:216–225.
   PubMed Web of Science ®Google Scholar
• Terazaki H, Ando Y, Nakamura M, Obayashi K, Misumi S, Shoji S, Yamashita S, Nakagawa K, Ishizaki T, Suhr O, et al Variant transthyretin in blood circulation can transverse the blood-cerebrospinal barrier: qualitative analyses of transthyretin metabolism in sequential liver transplantation. Transplantation 2001;72:296–299.
   PubMedGoogle Scholar
• Benson MD. Leptomeningeal amyloid and variant transthyretins. Am J Pathol 1996;148:351–354.
   PubMed Web of Science ®Google Scholar
• Said G, Planté-Bordeneuve V. Familial amyloid polyneuropathy a clinico-pathologic study. J Neurol Sci 2009;284:149–154.
   PubMed Web of Science ®Google Scholar
• Benson MD, Kluve-Beckerman B, Zeldenrust SR, Siesky AM, Bodenmiller DM, Showalter AD, Sloop KW. Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides. Muscle Nerve 2006;33:609–618.
   PubMed Web of Science ®Google Scholar
• Cossum PA, Sasmor H, Dellinger D, Truong L, Cummins L, Owens SR, Markham PM, Shea JP, Crooke S. Disposition of the 14C-labeled phosphorothioate oligonucleotide ISIS 2105 after intravenous administration to rats. J Pharm Sci Exp Ther 1993;267:1181–1190.
   PubMed Web of Science ®Google Scholar
• Smith RA, Miller TM, Yamanaka K, Monia BP, Condon TP, Hung G, Lobsiger CS, Ward CM, McAlonis-Downes M, Wei H, et al Antisense oligonucleotide therapy for neuro-degenerative disease. J Clin Invest 2006;116:2290–2296.
   PubMed Web of Science ®Google Scholar
• Holmgren G, Steen L, Ekstedt J, Groth C-G, Ericzon B-G, Eriksson S, Andersen O, Karlberg I, Nordén G, Nakazato M, et al Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet 1991;40:242–246.
   PubMed Web of Science ®Google Scholar
• Liepnieks JJ, Benson MD. Progression of cardiac amyloid deposition in hereditary transthyretin amyloidosis patients after liver transplantation. Amyloid 2007;14:277–282.
   PubMed Web of Science ®Google Scholar
• Munar-Qués M, Salva-Ladaria L, Mulet-Perera P, Solé M, López-Andreu FR, Saraviva JM. Vitreous amyloidosis after liver transplantation in patients with familial amyloid polyneuroapthy: ocular synthesis of mutant transthyretin. Amyloid Int J Exp Clin Invest 2000;7:266–269.
   PubMed Web of Science ®Google Scholar
• Ando Y, Terazaki H, Nakamura M, Ando E, Haraoka K, Yamashita T, Ueda M, Hirano K, Yamada M, Matsumoto K, et al A different amyloid formation mechanism: de novo oculoleptomeningeal amyloid deposits after liver transplantation. Transplantation 2004;77:345–349.
   PubMed Web of Science ®Google Scholar
• Yamashita T, Ando Y, Ueda M, Nakamura M, Okamoto S, Zeledon ME, Hirahara T, Hirai T, Ueda A, Misumi Y, et al Effect of liver transplantation on transthyretin Tyr114Cys-related cerebral amyloid angiopathy. Neurology 2008;70:123–128.
   PubMed Web of Science ®Google Scholar
• Dickson PW, Aldred AR, Marley PD, Guo-Fen T, Howlett GJ, Schreiber G. High prealbumin and transferrin mRNA levels in the choroid plexus of rat brain. Biochem Biophys Res Commun 1985;127:890–895.
   PubMed Web of Science ®Google Scholar
• Herbert J, Wilcox JN, Pham K-TC, Fremeau RT Jr, Zeviani M, Dwork A, Soprano DR, Makover A, Goodman DS, Zimmerman EA, et al Transthyretin: a choroid plexus-specific transport protein in human brain. Neurology 1986;36:900–911.
   PubMed Web of Science ®Google Scholar
• Li ZS, Dziegielewski KM, Saunders NR. Transthyretin distribution in the developing choroid plexus of the South American opossum (Monodelphis domestica). Cell Tissue Res 1997;287:621–624.
   PubMedGoogle Scholar