Kinetic stabilization of an oligomeric protein under physiological conditions demonstrated by a lack of subunit exchange: implications for transthyretin amyloidosis.

Apolipoprotein E4: a causative factor and therapeutic target in neuropathology, including Alzheimer's disease. Proc Natl Acad Sci U S A (2006) 5.00

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci U S A (2012) 2.27

The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J Mol Biol (2012) 1.70

Alpha-synuclein: from secretion to dysfunction and death. Cell Death Dis (2012) 1.25

Genistein, a natural product from soy, is a potent inhibitor of transthyretin amyloidosis. Proc Natl Acad Sci U S A (2005) 1.12

A substructure combination strategy to create potent and selective transthyretin kinetic stabilizers that prevent amyloidogenesis and cytotoxicity. J Am Chem Soc (2010) 1.02

Partitioning conformational intermediates between competing refolding and aggregation pathways: insights into transthyretin amyloid disease. Biochemistry (2005) 0.97

Characterization of the interaction of β-amyloid with transthyretin monomers and tetramers. Biochemistry (2010) 0.95

Aromatic sulfonyl fluorides covalently kinetically stabilize transthyretin to prevent amyloidogenesis while affording a fluorescent conjugate. J Am Chem Soc (2013) 0.88

Iodine atoms: a new molecular feature for the design of potent transthyretin fibrillogenesis inhibitors. PLoS One (2009) 0.87

Kinetic asymmetry of subunit exchange of homooligomeric protein as revealed by deuteration-assisted small-angle neutron scattering. Biophys J (2011) 0.86

Quantification of transthyretin kinetic stability in human plasma using subunit exchange. Biochemistry (2014) 0.85

Personalized medicine approach for optimizing the dose of tafamidis to potentially ameliorate wild-type transthyretin amyloidosis (cardiomyopathy). Amyloid (2015) 0.81

Drug Targeting of alpha-Synuclein Oligomerization in Synucleinopathies. Perspect Medicin Chem (2008) 0.80

Quantification of quaternary structure stability in aggregation-prone proteins under physiological conditions: the transthyretin case. Biochemistry (2014) 0.78

Mechanism of Action and Clinical Application of Tafamidis in Hereditary Transthyretin Amyloidosis. Neurol Ther (2016) 0.77

Protein fibrillation lag times during kinetic inhibition. Biophys J (2014) 0.76

Transthyretin variants with improved inhibition of β-amyloid aggregation. Protein Eng Des Sel (2016) 0.75

Adapting proteostasis for disease intervention. Science (2008) 13.22

Antibody domain exchange is an immunological solution to carbohydrate cluster recognition. Science (2003) 6.68

Opposing activities protect against age-onset proteotoxicity. Science (2006) 5.85

Biological and chemical approaches to diseases of proteostasis deficiency. Annu Rev Biochem (2009) 5.23

Semen-derived amyloid fibrils drastically enhance HIV infection. Cell (2007) 5.23

Hsp90 cochaperone Aha1 downregulation rescues misfolding of CFTR in cystic fibrosis. Cell (2006) 5.01

Therapeutic approaches to protein-misfolding diseases. Nature (2003) 4.57

Functional amyloid--from bacteria to humans. Trends Biochem Sci (2007) 4.50

Functional amyloid formation within mammalian tissue. PLoS Biol (2006) 3.92

Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology (2012) 3.48

Chemical and biological approaches synergize to ameliorate protein-folding diseases. Cell (2008) 3.11

Reduced IGF-1 signaling delays age-associated proteotoxicity in mice. Cell (2009) 2.87

The biological and chemical basis for tissue-selective amyloid disease. Cell (2005) 2.76

Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease. Proc Natl Acad Sci U S A (2002) 2.71

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis. Nat Chem Biol (2009) 2.70

Prevention of transthyretin amyloid disease by changing protein misfolding energetics. Science (2003) 2.63

An experimental survey of the transition between two-state and downhill protein folding scenarios. Proc Natl Acad Sci U S A (2008) 2.57

Context-dependent contributions of backbone hydrogen bonding to beta-sheet folding energetics. Nature (2004) 2.44

Structure-function-folding relationship in a WW domain. Proc Natl Acad Sci U S A (2006) 2.28

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci U S A (2012) 2.27

Native state kinetic stabilization as a strategy to ameliorate protein misfolding diseases: a focus on the transthyretin amyloidoses. Acc Chem Res (2005) 2.25

Sequence-dependent denaturation energetics: A major determinant in amyloid disease diversity. Proc Natl Acad Sci U S A (2002) 2.25

Chemical chaperones and permissive temperatures alter localization of Gaucher disease associated glucocerebrosidase variants. ACS Chem Biol (2006) 2.25

Tuning the free-energy landscape of a WW domain by temperature, mutation, and truncation. Proc Natl Acad Sci U S A (2003) 2.15

Tissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture. Proc Natl Acad Sci U S A (2004) 2.14

Transthyretin aggregation under partially denaturing conditions is a downhill polymerization. Biochemistry (2004) 2.08

Amyloid-β forms fibrils by nucleated conformational conversion of oligomers. Nat Chem Biol (2011) 2.08

Stress-independent activation of XBP1s and/or ATF6 reveals three functionally diverse ER proteostasis environments. Cell Rep (2013) 1.87

An adaptable standard for protein export from the endoplasmic reticulum. Cell (2007) 1.86

ALS mutants of human superoxide dismutase form fibrous aggregates via framework destabilization. J Mol Biol (2003) 1.82

Hydroxylated polychlorinated biphenyls selectively bind transthyretin in blood and inhibit amyloidogenesis: rationalizing rodent PCB toxicity. Chem Biol (2004) 1.76

Aβ induces astrocytic glutamate release, extrasynaptic NMDA receptor activation, and synaptic loss. Proc Natl Acad Sci U S A (2013) 1.73

Determinants for dephosphorylation of the RNA polymerase II C-terminal domain by Scp1. Mol Cell (2006) 1.71

The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J Mol Biol (2012) 1.70

Gaucher disease-associated glucocerebrosidases show mutation-dependent chemical chaperoning profiles. Chem Biol (2005) 1.68

Therapeutic strategies for human amyloid diseases. Nat Rev Drug Discov (2002) 1.63

Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses. Curr Opin Struct Biol (2010) 1.62

Potent kinetic stabilizers that prevent transthyretin-mediated cardiomyocyte proteotoxicity. Sci Transl Med (2011) 1.62

Elevated levels of oxidized cholesterol metabolites in Lewy body disease brains accelerate alpha-synuclein fibrilization. Nat Chem Biol (2006) 1.60

Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention. J Med Chem (2007) 1.58

Metabolite-initiated protein misfolding may trigger Alzheimer's disease. Proc Natl Acad Sci U S A (2004) 1.55

Pathogenesis of and therapeutic strategies to ameliorate the transthyretin amyloidoses. Curr Pharm Des (2008) 1.55

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol (2013) 1.54

Diflunisal analogues stabilize the native state of transthyretin. Potent inhibition of amyloidogenesis. J Med Chem (2004) 1.54

The core trisaccharide of an N-linked glycoprotein intrinsically accelerates folding and enhances stability. Proc Natl Acad Sci U S A (2009) 1.54

Amylin proprotein processing generates progressively more amyloidogenic peptides that initially sample the helical state. Biochemistry (2008) 1.51

Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants. Lab Invest (2004) 1.50

Engineering a beta-sheet protein toward the folding speed limit. J Phys Chem B (2005) 1.49

Partial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasis. PLoS Biol (2008) 1.42

Chemical and biological approaches for adapting proteostasis to ameliorate protein misfolding and aggregation diseases: progress and prognosis. Cold Spring Harb Perspect Biol (2011) 1.41

A fundamental protein property, thermodynamic stability, revealed solely from large-scale measurements of protein function. Proc Natl Acad Sci U S A (2012) 1.39

Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action. Angew Chem Int Ed Engl (2003) 1.39

Localized thermodynamic coupling between hydrogen bonding and microenvironment polarity substantially stabilizes proteins. Nat Struct Mol Biol (2009) 1.37

D18G transthyretin is monomeric, aggregation prone, and not detectable in plasma and cerebrospinal fluid: a prescription for central nervous system amyloidosis? Biochemistry (2003) 1.36

Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis. Amyloid (2006) 1.34

The pathway by which the tetrameric protein transthyretin dissociates. Biochemistry (2005) 1.32

Heparin accelerates gelsolin amyloidogenesis. Biochemistry (2006) 1.31

Pharmacologic chaperoning as a strategy to treat Gaucher disease. FEBS J (2007) 1.29

N-terminal domains elicit formation of functional Pmel17 amyloid fibrils. J Biol Chem (2009) 1.28

Protein native-state stabilization by placing aromatic side chains in N-glycosylated reverse turns. Science (2011) 1.27

Oxidative metabolites accelerate Alzheimer's amyloidogenesis by a two-step mechanism, eliminating the requirement for nucleation. Biochemistry (2005) 1.26

Dissociation of amyloid fibrils of alpha-synuclein and transthyretin by pressure reveals their reversible nature and the formation of water-excluded cavities. Proc Natl Acad Sci U S A (2003) 1.26

Quantification of the thermodynamically linked quaternary and tertiary structural stabilities of transthyretin and its disease-associated variants: the relationship between stability and amyloidosis. Biochemistry (2008) 1.26

Toward assessing the position-dependent contributions of backbone hydrogen bonding to beta-sheet folding thermodynamics employing amide-to-ester perturbations. J Am Chem Soc (2004) 1.26

Endoplasmic reticulum Ca2+ increases enhance mutant glucocerebrosidase proteostasis. Nat Chem Biol (2010) 1.26

Toward quantification of protein backbone-backbone hydrogen bonding energies: An energetic analysis of an amide-to-ester mutation in an alpha-helix within a protein. Protein Sci (2008) 1.26

The oxidative stress metabolite 4-hydroxynonenal promotes Alzheimer protofibril formation. Biochemistry (2007) 1.25

NMR solution structure of the isolated Apo Pin1 WW domain: comparison to the x-ray crystal structures of Pin1. Biopolymers (2002) 1.24

Probing the folding transition state structure of the villin headpiece subdomain via side chain and backbone mutagenesis. J Am Chem Soc (2009) 1.23

Synthesis, structure, and activity of diclofenac analogues as transthyretin amyloid fibril formation inhibitors. J Med Chem (2002) 1.22

Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res (2006) 1.20

Bisaryloxime ethers as potent inhibitors of transthyretin amyloid fibril formation. J Med Chem (2005) 1.19

Kinetic stabilization of an oligomeric protein by a single ligand binding event. J Am Chem Soc (2005) 1.18

X-ray structure of human acid-beta-glucosidase covalently bound to conduritol-B-epoxide. Implications for Gaucher disease. J Biol Chem (2005) 1.18

Biochemical and structural evaluation of highly selective 2-arylbenzoxazole-based transthyretin amyloidogenesis inhibitors. J Med Chem (2007) 1.17

Backbone-Backbone H-Bonds Make Context-Dependent Contributions to Protein Folding Kinetics and Thermodynamics: Lessons from Amide-to-Ester Mutations. Adv Protein Chem (2005) 1.16

Influence of the N-terminal domain on the aggregation properties of the prion protein. Protein Sci (2005) 1.16

Toward the molecular mechanism(s) by which EGCG treatment remodels mature amyloid fibrils. J Am Chem Soc (2013) 1.15

The effect of backbone cyclization on the thermodynamics of beta-sheet unfolding: stability optimization of the PIN WW domain. J Am Chem Soc (2002) 1.15

Kinetic stabilization of the native state by protein engineering: implications for inhibition of transthyretin amyloidogenesis. J Mol Biol (2005) 1.14

Discovery and characterization of a mammalian amyloid disaggregation activity. Protein Sci (2010) 1.13

Temporal requirements of insulin/IGF-1 signaling for proteotoxicity protection. Aging Cell (2009) 1.13

Genistein, a natural product from soy, is a potent inhibitor of transthyretin amyloidosis. Proc Natl Acad Sci U S A (2005) 1.12

Synthesis and characterization of potent bivalent amyloidosis inhibitors that bind prior to transthyretin tetramerization. J Am Chem Soc (2003) 1.12

Small molecule oxidation products trigger disease-associated protein misfolding. Acc Chem Res (2006) 1.11

A biomimetic synthesis of thiazolines using hexaphenyloxodiphosphonium trifluoromethanesulfonate. Angew Chem Int Ed Engl (2003) 1.11

Energetic characteristics of the new transthyretin variant A25T may explain its atypical central nervous system pathology. Lab Invest (2003) 1.10

Recombinant expression, purification, and comparative characterization of torsinA and its torsion dystonia-associated variant Delta E-torsinA. Biochemistry (2003) 1.10

Toward optimization of the linker substructure common to transthyretin amyloidogenesis inhibitors using biochemical and structural studies. J Med Chem (2008) 1.10

Structural insight into pH-induced conformational changes within the native human transthyretin tetramer. J Mol Biol (2008) 1.08

Cys10 mixed disulfides make transthyretin more amyloidogenic under mildly acidic conditions. Biochemistry (2003) 1.08

Biochemical properties of highly neuroinvasive prion strains. PLoS Pathog (2012) 1.07

Evaluating beta-turn mimics as beta-sheet folding nucleators. Proc Natl Acad Sci U S A (2009) 1.07

Amide-to-E-olefin versus amide-to-ester backbone H-bond perturbations: Evaluating the O-O repulsion for extracting H-bond energies. J Am Chem Soc (2006) 1.06

Synthesis of all nineteen appropriately protected chiral alpha-hydroxy acid equivalents of the alpha-amino acids for Boc solid-phase depsi-peptide synthesis. Org Lett (2004) 1.05

Hydration and packing are crucial to amyloidogenesis as revealed by pressure studies on transthyretin variants that either protect or worsen amyloid disease. J Mol Biol (2003) 1.05

Human-murine transthyretin heterotetramers are kinetically stable and non-amyloidogenic. A lesson in the generation of transgenic models of diseases involving oligomeric proteins. J Biol Chem (2007) 1.04

Site-specific modification of Alzheimer's peptides by cholesterol oxidation products enhances aggregation energetics and neurotoxicity. Proc Natl Acad Sci U S A (2009) 1.04

Retention of misfolded mutant transthyretin by the chaperone BiP/GRP78 mitigates amyloidogenesis. J Mol Biol (2005) 1.04