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Article

Emerging novel concept of chaperone therapies for protein misfolding diseases.

Proceedings of the Japan Academy. Series B, Physical and biological sciences - 1 Jan 2014

Suzuki Yoshiyuki

Abstract excerpt

Chaperone therapy is a newly developed molecular therapeutic approach to protein misfolding diseases. Among them we found unstable mutant enzyme proteins in a few lysosomal diseases, resulting in rapid intracellular degradation and loss of function. Active-site binding low molecular competitive inhibitors (chemical chaperones) paradoxically stabilized and enhanced the enzyme activity in somatic cells by...

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Identifiers

PMID: 24814990
DOI: 10.2183/pjab.90.145
PMCID: PMC4104511

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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