Therapeutic targeting of misfolding and conformational change in α1-antitrypsin deficiency (Q35214289)

7 August 2017

Therapeutic targeting of misfolding and conformational change in α1-antitrypsin deficiency (English)

1 reference

7 August 2017

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25068987%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

author name string

Mun Peak Nyon

series ordinal

1

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31 May 2020

publication date

1 June 2014

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Future Medicinal Chemistry

7 August 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25068987%20AND%20SRC:MED&resulttype=core&format=json

31 May 2020

volume

6

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31 May 2020

issue

9

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31 May 2020

page(s)

1047-1065

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The serpins are an expanding superfamily of structurally similar but functionally diverse proteins. Evolution, mechanism of inhibition, novel functions, and a revised nomenclature

31 May 2020

cites work

1 reference

The molecular and cellular pathology of α₁-antitrypsin deficiency.

21 January 2018

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Prevalence of α1-antitrypsin deficiency alleles PI*S and PI*Z worldwide and effective screening for each of the five phenotypic classes PI*MS, PI*MZ, PI*SS, PI*SZ, and PI*ZZ: a comprehensive review

21 January 2018

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Genetic epidemiology of alpha-1 antitrypsin deficiency in southern Europe: France, Italy, Portugal and Spain.

21 January 2018

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Association of IREB2 and CHRNA3 polymorphisms with airflow obstruction in severe alpha-1 antitrypsin deficiency

21 January 2018

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Conceptual advances in the pathogenesis and treatment of childhood metabolic liver disease.

21 January 2018

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The mechanism of Z alpha 1-antitrypsin accumulation in the liver

21 January 2018

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Cleavage of CXCR1 on neutrophils disables bacterial killing in cystic fibrosis lung disease

21 January 2018

1 reference

Neutrophil elastase-mediated lung disease

21 January 2018

1 reference

Lung polymers in Z alpha1-antitrypsin deficiency-related emphysema

21 January 2018

1 reference

Polymers of Z alpha1-antitrypsin co-localize with neutrophils in emphysematous alveoli and are chemotactic in vivo

21 January 2018

1 reference

Polymers of alpha(1)-antitrypsin are chemotactic for human neutrophils: a new paradigm for the pathogenesis of emphysema

21 January 2018

1 reference

Mechanisms of emphysema in alpha1-antitrypsin deficiency: molecular and cellular insights

21 January 2018

1 reference

A surprising new protein superfamily containing ovalbumin, antithrombin-III, and alpha 1-proteinase inhibitor

21 January 2018

1 reference

Implications of the three-dimensional structure of alpha 1-antitrypsin for structure and function of serpins

21 January 2018

1 reference

Conformational pathology of the serpins: themes, variations, and therapeutic strategies

21 January 2018

1 reference

Association between conformational mutations in neuroserpin and onset and severity of dementia

21 January 2018

1 reference

Tissue-type plasminogen activator and neuroserpin: a well-balanced act in the nervous system?

21 January 2018

1 reference

Polymers and inflammation: disease mechanisms of the serpinopathies

21 January 2018

1 reference

A novel monoclonal antibody to characterize pathogenic polymers in liver disease associated with alpha1-antitrypsin deficiency.

21 January 2018

1 reference

Defining the mechanism of polymerization in the serpinopathies

21 January 2018

1 reference

Structural dynamics associated with intermediate formation in an archetypal conformational disease.

21 January 2018

1 reference

Structure of a serpin-protease complex shows inhibition by deformation

21 January 2018

1 reference

Antithrombin-S195A factor Xa-heparin structure reveals the allosteric mechanism of antithrombin activation

21 January 2018

1 reference

Wild-type alpha 1-antitrypsin is in the canonical inhibitory conformation.

21 January 2018

1 reference

Targeting serpins in high-throughput and structure-based drug design

21 January 2018

1 reference

Conformational changes and disease--serpins, prions and Alzheimer's.

21 January 2018

1 reference

Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases

21 January 2018

1 reference

Molecular gymnastics: serpin structure, folding and misfolding

21 January 2018

1 reference

Human alpha 1-proteinase inhibitor. Crystal structure analysis of two crystal modifications, molecular model and preliminary analysis of the implications for function

21 January 2018

1 reference

Hormone binding globulins undergo serpin conformational change in inflammation

21 January 2018

1 reference

How changes in affinity of corticosteroid-binding globulin modulate free cortisol concentration

21 January 2018

1 reference

What do dysfunctional serpins tell us about molecular mobility and disease?

21 January 2018

1 reference

Inactive conformation of the serpin alpha(1)-antichymotrypsin indicates two-stage insertion of the reactive loop: implications for inhibitory function and conformational disease

21 January 2018

1 reference

Molecular basis of α1-antitrypsin deficiency revealed by the structure of a domain-swapped trimer

21 January 2018

1 reference

Crystal structure of a stable dimer reveals the molecular basis of serpin polymerization

21 January 2018

1 reference

Therapeutic target-site variability in α1-antitrypsin characterized at high resolution

21 January 2018

1 reference

Topography of a 2.0 Å structure of α1-antitrypsin reveals targets for rational drug design to prevent conformational disease

21 January 2018

1 reference

Preventing serpin aggregation: The molecular mechanism of citrate action upon antitrypsin unfolding

21 January 2018

1 reference

On the size of the active site in proteases. I. Papain

21 January 2018

1 reference

The native strains in the hydrophobic core and flexible reactive loop of a serine protease inhibitor: crystal structure of an uncleaved alpha1-antitrypsin at 2.7 A

21 January 2018

1 reference

Regulation of protein function by native metastability

21 January 2018

1 reference

Targeting a surface cavity of alpha 1-antitrypsin to prevent conformational disease

21 January 2018

1 reference

Kinetic instability of the serpin Z alpha1-antitrypsin promotes aggregation.

21 January 2018

1 reference

Probing the equilibrium denaturation of the serpin alpha(1)-antitrypsin with single tryptophan mutants; evidence for structure in the urea unfolded state

21 January 2018

1 reference

Probing the unfolding pathway of alpha1-antitrypsin.

21 January 2018

1 reference

Folding mechanism of the metastable serpin α1-antitrypsin

21 January 2018

1 reference

The structural basis of serpin polymerization studied by hydrogen/deuterium exchange and mass spectrometry

21 January 2018

1 reference

The serpinopathies studying serpin polymerization in vivo

21 January 2018

1 reference

A 2.6 A structure of a serpin polymer and implications for conformational disease

21 January 2018

1 reference

Reactive centre loop mutants of α-1-antitrypsin reveal position-specific effects on intermediate formation along the polymerization pathway

21 January 2018

1 reference

Elucidation of the molecular logic by which misfolded alpha 1-antitrypsin is preferentially selected for degradation.

21 January 2018

1 reference

The proteasome participates in degradation of mutant alpha 1-antitrypsin Z in the endoplasmic reticulum of hepatoma-derived hepatocytes

21 January 2018

1 reference

Intracellular processing of alpha1-antitrypsin

21 January 2018

1 reference

Sec61p is part of the endoplasmic reticulum-associated degradation machinery

21 January 2018

1 reference

Derlin-1 is a rhomboid pseudoprotease required for the dislocation of mutant α-1 antitrypsin from the endoplasmic reticulum

21 January 2018

1 reference

SEL1L protein critically determines the stability of the HRD1-SEL1L endoplasmic reticulum-associated degradation (ERAD) complex to optimize the degradation kinetics of ERAD substrates

21 January 2018

1 reference

Stimulation of ERAD of misfolded null Hong Kong alpha1-antitrypsin by Golgi alpha1,2-mannosidases

21 January 2018

1 reference

Endoplasmic reticulum stress signaling in disease

21 January 2018

1 reference

The molecular aetiology of the serpinopathies

21 January 2018

1 reference

Endoplasmic reticulum polymers impair luminal protein mobility and sensitize to cellular stress in alpha1-antitrypsin deficiency

21 January 2018

1 reference

Accumulation of mutant alpha1-antitrypsin Z in the endoplasmic reticulum activates caspases-4 and -12, NFkappaB, and BAP31 but not the unfolded protein response.

21 January 2018

1 reference

Endoplasmic reticulum-associated degradation (ERAD) and autophagy cooperate to degrade polymerogenic mutant serpins

21 January 2018

1 reference

Neuroserpin polymers activate NF-kappaB by a calcium signaling pathway that is independent of the unfolded protein response.

21 January 2018

1 reference

The ER-overload response: activation of NF-kappa B.

21 January 2018

1 reference

Evidence for unfolded protein response activation in monocytes from individuals with alpha-1 antitrypsin deficiency

21 January 2018

1 reference

Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of α1-antitrypsin deficiency

21 January 2018

1 reference

Small molecules block the polymerization of Z alpha1-antitrypsin and increase the clearance of intracellular aggregates

21 January 2018

1 reference

Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells.

21 January 2018

1 reference

Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette.

21 January 2018

1 reference

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

21 January 2018

1 reference

A novel interaction between aging and ER overload in a protein conformational dementia

21 January 2018

1 reference

Natural genetic variation determines susceptibility to aggregation or toxicity in a C. elegans model for polyglutamine disease

21 January 2018

1 reference

Utility of an improved model of amyloid-beta (Aβ₁₋₄₂) toxicity in Caenorhabditis elegans for drug screening for Alzheimer's disease

21 January 2018

1 reference

Automated high-content live animal drug screening using C. elegans expressing the aggregation prone serpin α1-antitrypsin Z

21 January 2018

1 reference

Modeling molecular and cellular aspects of human disease using the nematode Caenorhabditis elegans

21 January 2018

1 reference

Hypothesis-based RNAi screening identifies neuroprotective genes in a Parkinson's disease model

21 January 2018

1 reference

Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice

21 January 2018

1 reference

An autophagy-enhancing drug promotes degradation of mutant alpha1-antitrypsin Z and reduces hepatic fibrosis.

21 January 2018

1 reference

Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: A potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency

21 January 2018

1 reference

Rapamycin reduces intrahepatic alpha-1-antitrypsin mutant Z protein polymers and liver injury in a mouse model

21 January 2018

1 reference

Oxidation of Z α1-antitrypsin by cigarette smoke induces polymerization: a novel mechanism of early-onset emphysema.

21 January 2018

1 reference

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins

21 January 2018

1 reference

Oxidative stress contributes to liver damage in a murine model of alpha-1-antitrypsin deficiency.

21 January 2018

1 reference

Smoking, lung function, and alpha 1-antitrypsin deficiency.

21 January 2018

1 reference

The Copenhagen National Lung Transplant Group: survival after single lung, double lung, and heart-lung transplantation

21 January 2018

1 reference

Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema

21 January 2018

1 reference

New Findings in PiZZ alpha1-antitrypsin deficiency-related panniculitis. Demonstration of skin polymers and high dosing requirements of intravenous augmentation therapy

21 January 2018

1 reference

alpha 1-antitrypsin deficiency-associated panniculitis: resolution with intravenous alpha 1-antitrypsin administration and liver transplantation

21 January 2018

1 reference

Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency.

21 January 2018

1 reference

Augmentation therapy for alpha-1 antitrypsin deficiency: towards a personalised approach

21 January 2018

1 reference

Augmentation therapy in alpha-1 antitrypsin deficiency: advances and controversies

21 January 2018

1 reference

Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice.

21 January 2018

1 reference

Proteostasis strategies for restoring alpha1-antitrypsin deficiency

21 January 2018

1 reference

Chemical chaperones: a pharmacological strategy for disorders of protein folding and trafficking

21 January 2018

1 reference

Sugar and alcohol molecules provide a therapeutic strategy for the serpinopathies that cause dementia and cirrhosis

21 January 2018

1 reference

Lack of effect of oral 4-phenylbutyrate on serum alpha-1-antitrypsin in patients with alpha-1-antitrypsin deficiency: a preliminary study

21 January 2018

1 reference

Small-molecule peptides inhibit Z alpha1-antitrypsin polymerization

21 January 2018

1 reference

Inhibiting polymerization: new therapeutic strategies for Z alpha1-antitrypsin-related emphysema.

21 January 2018

1 reference

6-mer peptide selectively anneals to a pathogenic serpin conformation and blocks polymerization. Implications for the prevention of Z alpha(1)-antitrypsin-related cirrhosis

21 January 2018

1 reference

In silico assessment of potential druggable pockets on the surface of α1-antitrypsin conformers

21 January 2018

1 reference

Crystallographic and Cellular Characterisation of Two Mechanisms Stabilising the Native Fold of α1-Antitrypsin: Implications for Disease and Drug Design

21 January 2018

1 reference

Implications for function and therapy of a 2.9 A structure of binary-complexed antithrombin

21 January 2018

1 reference

A novel model and molecular therapy for Z alpha-1 antitrypsin deficiency.

21 January 2018

1 reference

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency

21 January 2018

1 reference

Gene-based therapy for alpha-1 antitrypsin deficiency

21 January 2018

1 reference

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.

21 January 2018

1 reference

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

21 January 2018

1 reference

A Kinetic Mechanism for the Polymerization of α1-Antitrypsin

21 January 2018

1 reference