References
- BuxbaumJNTransthyretin and the Transthyretin AmyloidosesUverskyVNFinkAProtein Misfolding, Aggregation, and Conformational DiseasesSanta Cruz, CASpringer2007259283
- ReixachNDeechongkitSJiangXKellyJWBuxbaumJNTissue damage in the amyloidoses: Transthyretin monomers and non-native oligomers are the major cytotoxic species in tissue cultureProc Natl Acad Sci U S A200410192817282214981241
- WhiteJTKellyJWSupport for the multigenic hypothesis of amyloidosis: the binding stoichiometry of retinol-binding protein, vitamin A, and thyroid hormone influences transthyretin amyloidogenicity in vitroProc Natl Acad Sci U S A20019823130191302411687657
- SchreiberGSouthwellBRRichardsonSJHormone delivery systems to the brain – transthyretinExp Clin Endocrinol19951037580
- SchreiberGThe evolution of transthyretin synthesis in the choroid plexusClin Chem Lab Med200240121200121012553420
- RedzicZBSegalMBThe structure of the choroid plexus and the physiology of the choroid plexus epitheliumAdv Drug Deliv Rev200456121695171615381330
- CostaRHGraysonDRDarnellJEMultiple hepatocyte-enriched nuclear factors function in the regulation of transthyretin and α1-antitrypsin genesMol Cell Biol19899141514252786140
- SamadaniUCostaRHThe transcriptional activator hepatocyte nuclear factor 6 regulates liver gene expressionMol Cell Biol19961611627362848887657
- CostaRHGraysonDRSite-directed mutagenesis of hepatocyte nuclear factor (HNF) binding sites in the mouse transthyretin (TTR) promoter reveal synergistic interactions with its enhancer regionNucleic Acids Res19911915413941451870969
- QianXSamadaniUPorcellaACostaRHDecreased expression of hepatocyte nuclear factor 3 alpha during the acute-phase response influences transthyretin gene transcriptionMol Cell Biol1995153136413767862129
- DicksonPWAldredARMarleyPDBannisterDSchreiberGRat choroid plexus specializes in the synthesis and the secretion of transthyretin (prealbumin). Regulation of transthyretin synthesis in choroid plexus is independent from that in liverJ Biol Chem19862618347534783949774
- LiXBuxbaumJNTransthyretin and the brain re-visited: is neuronal synthesis of transthyretin protective in Alzheimer’s disease?Mol Neurodegener201167922112803
- RefaiEDekkiNYangSNTransthyretin constitutes a functional component in pancreatic beta-cell stimulus-secretion couplingProc Natl Acad Sci U S A200510247170201702516286652
- ChristmanssonLBetsholtzCGustavssonÅJohanssonBSlettenKWestermarkPThe transthyretin cDNA sequence is normal in transthyretin-derived senile systemic amyloidosisFEBS Lett19912811771802015890
- SteinKStorkelSLinkeRPGoebelHHChemical heterogeneity of amyloid in the carpal tunnel syndromeVirchows Arch A Pathol Anat Histopathol1987412137453120402
- RockenCSaegerWLinkeRPGastrointestinal amyloid deposits in old agePathol Res Pract19941906416497808962
- HurshmanARWhiteJTPowersETKellyJWTransthyretin aggregation under partially denaturing conditions is a downhill polymerizationBiochemistry200443237365738115182180
- ColonWKellyJWPartial denaturation of transthyretin is sufficient for amyloid fibril formation in vitroBiochemistry19923136865486601390650
- SekijimaYWisemanRLMattesonJThe biological and chemical basis for tissue-selective amyloid diseaseCell20051211738515820680
- SousaACoelhoTSequeirosJParental transmission and age of onset in Familial Amyloidotic Polyneuropathy (Portuguese type)Amyloid and AmyloidosisKluwer1991691693
- CoelhoTCarvalhoMSaraivaMAlvesIAlmeidaMRCostaPPA strikingly benign evolution of FAP in an individual found to be a compound heterozygote for two TTR mutations: TTR MET 30 and TTR MET 119J Rheumatol199320179
- JacobsonDRMcFarlinDEKaneIBuxbaumJNTransthyretin Pro55, a variant associated with early-onset, aggressive, diffuse amyloidosis with cardiac and neurologic involvementHum Genet19928933533561351039
- SousaACoelhoTLobatoLSequeirosJAnticipation of Age of Onset in Familial Amyloidotic Polyneuropathy (Portuguese type)Philadelphia, PAKluwer Academic Publishers1990694697
- AndradeCA peculiar form of peripheral neuropathy. Familial atypical generalized amyloidosis with special involvement of the peripheral nervesBrain19527540842712978172
- CostaPPFigueiraASBravoFRAmyloid fibril protein related to prealbumin in familial amyloidotic polyneuropathyProc Natl Acad Sci U S A19787544994503279930
- ArakiSMawatariSOhtaMNakajimaAKuroiwaYPolyneuritic amyloidosis in a Japanese familyArch Neurol19681865936025652991
- DruggeUAnderssonRChizariFFamilial amyloidotic polyneuropathy in Sweden: a pedigree analysisJ Med Genet1993303883928100581
- YoshiokaKFuruyaHSasakiHSaraivaMJMCostaPPSakakiYHaplotype analysis of familial amyloidotic polyneuropathy. Evidence for multiple origins of the Val-Met mutation most common to the diseaseHum Genet1989829132714785
- HolmgrenGCostaPMAnderssonCGeographical distribution of TTR met30 carriers in northern Sweden: discrepancy between carrier frequency and prevalence rateJ Med Genet1994313513548064809
- HardellLHolmgrenGSteenLFredriksonMAxelsonOOccupational and other risk factors for clinically overt familial amyloid polyneuropathyEpidemiology199565986018589090
- Plante-BordeneuveVSaidGFamilial amyloid polyneuropathyLancet Neurol201110121086109722094129
- SuhrOBEriczonBGFrimanSLong-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type)Liver Transpl20028978779412200779
- HolmgrenGEriczonBGGrothCGClinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosisLancet1993341111311168097803
- WilczekHELarssonMEriczonBGLong-term data from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR)Amyloid201118Suppl 1188190
- SuhrODanielssonAHolmgrenGSteenLMalnutrition and gastrointestinal dysfunction as prognostic factors for survival in familial amyloidotic polyneuropathyJ Intern Med199423554794858182405
- YazakiMTokudaTNakamuraACardiac amyloid in patients with familial amyloidotic polyneuropathy consists of abundant wild-type transthyretinBiochem Biophys Res Commun200027470270610924339
- BarreirosAPPostFHoppe-LotichiusMLiver transplantation and combined liver-heart transplantation in patients with familial amyloid polyneuropathy: a single-center experienceLiver Transpl201016331432320209591
- AndoYEriczonBGSuhrOBTashimaKAndoMReuse of a Japanese familial amyloidotic polyneuropathy patient’s liver for a cancer patient: the domino liver transplantation procedureIntern Med199736118479392364
- MonteiroEPerdigotoRFurtadoALLiver transplantation for familial amyloid polyneuropathyHepatogastroenterology19984523137513809840068
- StangouAJHeatonNDHawkinsPNTransmission of systemic transthyretin amyloidosis by means of domino liver transplantationN Engl J Med200535222235615930432
- MiroyGJLaiZLashuelHAPetersonSAStrangCKellyJWInhibiting transthyretin amyloid fibril formation via protein stabilizationProc Natl Acad Sci U S A19969315051150568986762
- BauresPWOzaVBPetersonSAKellyJWSynthesis and evaluation of inhibitors of transthyretin amyloid formation based on the non-steroidal anti-inflammatory drug, flufenamic acidBioorg Med Chem1999771339134710465408
- Adamski-WernerSLPalaninathanSKSacchettiniJCKellyJWDiflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesisJ Med Chem200447235537414711308
- BerkJLDyckPJObiciLThe diflunisal trial: update on study drug tolerance and disease progressionAmyloid201118Suppl 119119222080762
- ReixachNFossTRSantelliEPascualJKellyJWBuxbaumJNHuman-murine transthyretin heterotetramers are kinetically stable and non-amyloidogenic. A lesson in the generation of transgenic models of diseases involving oligomeric proteinsJ Biol Chem200828342098210718006495
- JohnsonSMConnellySFearnsCPowersETKellyJWThe transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drugJ Mol Biol201218520322244854
- BulawaCEConnellySDevitMTafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascadeProc Natl Acad Sci U S A2012109249629963422645360
- CoelhoTMaiaLFMartins da SilvaATafamidis for transthyretin familial amyloid polyneuropathyNeurology20127918
- DyckPJLitchyWJDaubeJRIndividual attributes versus composite scores of nerve conduction abnormality: sensitivity, reproducibility, and concordance with impairmentMuscle Nerve200327220221012548528
- VinikEJHayesRPOglesbyAThe development and validation of the Norfolk QOL-DN, a new measure of patients’ perception of the effects of diabetes and diabetic neuropathyDiabet Technol Ther200573497508
- KimDHZeldenrustSRLowPADyckPJQuantitative sensation and autonomic test abnormalities in transthyretin amyloidosis polyneuropathyMuscle Nerve200940336337019618439
- FeinsteinARClinical Epidemiology: The Architecture of Clinical ResearchPhiladelphia, PAW.B. Saunders Company1985
- KatzRJillapalliDLuanJPeripheral and central nervous System Drugs Advisory Committee Meeting, Background package 24 May 2012 Available at www.fda.gov/downloads/AdvisorycommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdviso-ryCommittee/UCM304830.pdf
- YamashitaTAndoYOkamotoSLong-term survival after liver transplantation in patients with familial amyloid polyneuropathyNeurology201278963764322345221
- KristenAVLehrkeSBussSGreen tea halts progression of cardiac transthyretin amyloidosis: an observational reportClin Res Cardiol20121011080581322584381
- ObiciLCorteseALozzaADoxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II studyAmyloid201219Suppl 1343622551192
- SaundersAMStrittmatterWJSchmechelDAssociation of apolipoprotein E allele epsilon 4 with late-onset familial and sporadic Alzheimer’s diseaseNeurology199343146714728350998
- BottoMHawkinsPNBickerstaffMCAmyloid deposition is delayed in mice with targeted deletion of the serum amyloid P component geneNat Med1997388558599256275
- PepysMBHerbertJHutchinsonWLTargeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosisNature2002417688625425912015594
- AlvarezRBorlandTChenQALN-TTR, an RNAi therapeutic for the treatment of Transthyretin-mediated amyloidosisAmyloid201017s15152
- BensonMDPandeySWitchellDAntisense oligonucleotide therapy for TTR amyloidosisAmyloid201118Suppl 15521838431