(Q34285075)

1 August 2017

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title

Therapeutic approaches to protein-misfolding diseases (English)

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1 August 2017

1

Fred E Cohen

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1 August 2017

Jeffery W Kelly

2

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1 August 2017

language of work or name

English

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publication date

1 December 2003

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1 August 2017

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1 August 2017

volume

426

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1 August 2017

issue

6968

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1 August 2017

page(s)

905-909

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Principles that govern the folding of protein chains

1 August 2017

cites work

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Alpha1-antitrypsin deficiency--a model for conformational diseases

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Amyloid fibrillogenesis: themes and variations

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Alternative conformations of amyloidogenic proteins govern their behavior

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A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity

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Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease

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Pharmacological chaperones rescue cell-surface expression and function of misfolded V2 vasopressin receptor mutants

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Ligands act as pharmacological chaperones and increase the efficiency of delta opioid receptor maturation

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Structure-activity relations of successful pharmacologic chaperones for rescue of naturally occurring and manufactured mutants of the gonadotropin-releasing hormone receptor

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Pharmacological chaperone-mediated in vivo folding and stabilization of the P23H-opsin mutant associated with autosomal dominant retinitis pigmentosa

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The binding site for channel blockers that rescue misprocessed human long QT syndrome type 2 ether-a-gogo-related gene (HERG) mutations

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Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

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Improvement in cardiac function in the cardiac variant of Fabry's disease with galactose-infusion therapy

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Benzoflavone activators of the cystic fibrosis transmembrane conductance regulator: towards a pharmacophore model for the nucleotide-binding domain

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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

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Sequence-dependent denaturation energetics: A major determinant in amyloid disease diversity

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Prevention of transthyretin amyloid disease by changing protein misfolding energetics.

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Transthyretin slowly exchanges subunits under physiological conditions: A convenient chromatographic method to study subunit exchange in oligomeric proteins.

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Mechanism of rescue of common p53 cancer mutations by second-site suppressor mutations

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Therapeutic strategies for human amyloid diseases

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Support for the multigenic hypothesis of amyloidosis: the binding stoichiometry of retinol-binding protein, vitamin A, and thyroid hormone influences transthyretin amyloidogenicity in vitro

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Acridine and phenothiazine derivatives as pharmacotherapeutics for prion disease

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Potent inhibition of scrapie prion replication in cultured cells by bis-acridines

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Antimalarial drug quinacrine binds to C-terminal helix of cellular prion protein

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Antibodies inhibit prion propagation and clear cell cultures of prion infectivity

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Monoclonal antibodies inhibit prion replication and delay the development of prion disease

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Prevention of scrapie pathogenesis by transgenic expression of anti-prion protein antibodies

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Anti-aggregating antibodies, a new approach towards treatment of conformational diseases

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7 January 2021

Prion propagation in mice expressing human and chimeric PrP transgenes implicates the interaction of cellular PrP with another protein

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7 January 2021

The antisense approach in amyloid light chain amyloidosis: identification of monoclonal Ig and inhibition of its production by antisense oligonucleotides in in vitro and in vivo models

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Peptide inhibitors of beta amyloid aggregation

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Amyloid as a natural product

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7 January 2021

Diffusible, nonfibrillar ligands derived from Abeta1-42 are potent central nervous system neurotoxins

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7 January 2021

Common structure of soluble amyloid oligomers implies common mechanism of pathogenesis

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7 January 2021

Naturally secreted oligomers of amyloid beta protein potently inhibit hippocampal long-term potentiation in vivo

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7 January 2021

Secretase as a target for Alzheimer's disease

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Beta-secretase (BACE) as a drug target for Alzheimer's disease

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7 January 2021

Seeding "one-dimensional crystallization" of amyloid: a pathogenic mechanism in Alzheimer's disease and scrapie?

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7 January 2021

The generation and characterization of potentially therapeutic Abeta antibodies in mice: differences according to strain and immunization protocol

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7 January 2021

Neuropathology of human Alzheimer disease after immunization with amyloid-beta peptide: a case report.

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7 January 2021

Partial denaturation of transthyretin is sufficient for amyloid fibril formation in vitro

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7 January 2021

Amyloid fibrils from muscle myoglobin

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7 January 2021

Folding of prion protein to its native alpha-helical conformation is under kinetic control

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7 January 2021

Identifiers

DOI

10.1038/NATURE02265

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Dimensions Publication ID

1 August 2017

1053582289

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1 reference