Small molecules block the polymerization of Z alpha1-antitrypsin and increase the clearance of intracellular aggregates (Q24645055)

1

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Russell L Phillips

2

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S Adrian Saldanha

3

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Bibek Gooptu

4

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Sarah C Leigh Brown

5

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Daniel J Termine

6

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Arash M Shirvani

7

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Ying Wu

8

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Richard N Sifers

9

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David A Lomas

Journal of Medicinal Chemistry

11

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language of work or name

English

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publication date

1 November 2007

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published in

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volume

50

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issue

22

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page(s)

5357-63

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cites work

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Structure of a serpin-protease complex shows inhibition by deformation

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

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

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Mutant Neuroserpin (S49P) that causes familial encephalopathy with neuroserpin inclusion bodies is a poor proteinase inhibitor and readily forms polymers in vitro

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The selective advantage of alpha1-antitrypsin deficiency

7 April 2017

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Quantitative isolation of alphalAT mutant Z protein polymers from human and mouse livers and the effect of heat

27 September 2017

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Differential detection of PAS-positive inclusions formed by the Z, Siiyama, and Mmalton variants of alpha1-antitrypsin.

27 September 2017

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

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Antithrombin Phe229Leu: a new homozygous variant leading to spontaneous antithrombin polymerization in vivo associated with severe childhood thrombosis.

27 September 2017

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Organizational diversity among distinct glycoprotein endoplasmic reticulum-associated degradation programs

27 September 2017

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

27 September 2017

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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

27 September 2017

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Inhibition of intracellular degradation increases secretion of a mutant form of alpha1-antitrypsin associated with profound deficiency

27 September 2017

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A hinge region mutation in C1-inhibitor (Ala436-->Thr) results in nonsubstrate-like behavior and in polymerization of the molecule.

27 September 2017

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Biased probability Monte Carlo conformational searches and electrostatic calculations for peptides and proteins

27 September 2017

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A lag in intracellular degradation of mutant alpha 1-antitrypsin correlates with the liver disease phenotype in homozygous PiZZ alpha 1-antitrypsin deficiency

27 September 2017

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

27 September 2017

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COOH-terminal substitutions in the serpin C1 inhibitor that cause loop overinsertion and subsequent multimerization

27 September 2017

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Optimal protocol and trajectory visualization for conformational searches of peptides and proteins

27 September 2017

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Identification of a 4-mer Peptide Inhibitor that Effectively Blocks the Polymerization of Pathogenic Z α1-Antitrypsin

30 May 2018

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Alpha 1-antitrypsin polymerization: a fluorescence correlation spectroscopic study.

28 November 2018

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

28 November 2018

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

28 November 2018

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Nucleation of alpha 1-antichymotrypsin polymerization

28 November 2018

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

28 November 2018

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Pathogenic α1-Antitrypsin Polymers Are Formed by Reactive Loop-β-Sheet A Linkage

28 November 2018

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Mutations which impede loop/sheet polymerization enhance the secretion of human alpha 1-antitrypsin deficiency variants.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2631427

Liver Disease in Alpha1-Antitrypsin Deficiency Detected by Screening of 200,000 Infants

28 November 2018

1 reference

12 December 2020

Risk of cirrhosis and primary liver cancer in alpha 1-antitrypsin deficiency

1 reference

12 December 2020

Effect of the Z mutation on the physical and inhibitory properties of alpha 1-antitrypsin

1 reference

12 December 2020

Degradation of a mutant secretory protein, alpha1-antitrypsin Z, in the endoplasmic reticulum requires proteasome activity

1 reference

12 December 2020

A Kinetic Mechanism for the Polymerization of α1-Antitrypsin

1 reference

12 December 2020

Intracellular inclusions containing mutant alpha1-antitrypsin Z are propagated in the absence of autophagic activity

1 reference

12 December 2020

Processing by endoplasmic reticulum mannosidases partitions a secretion-impaired glycoprotein into distinct disposal pathways

1 reference

12 December 2020

Retention of mutant alpha(1)-antitrypsin Z in endoplasmic reticulum is associated with an autophagic response

1 reference

12 December 2020

Prevention of polymerization of M and Z alpha1-Antitrypsin (alpha1-AT) with trimethylamine N-oxide. Implications for the treatment of alpha1-at deficiency

1 reference

12 December 2020

Identifiers

DOI

10.1021/JM070687Z

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1353392

1353392

1 reference

1 reference

1 reference