Abstract
Chaperone therapy was introduced first as a new molecular therapeutic approach to
lysosomal diseases. In a recent article, I reviewed the development of chaperone therapy
mainly for lysosomal diseases. Then, more data have been collected particularly on
non-lysosomal protein misfolding diseases. In this short review, I propose the concept
of chaperone therapy to be classified into two different therapeutic approaches, for
pH-dependent lysosomal, and pH-independent non-lysosomal protein misfolding diseases.
The concept of lysosomal chaperone therapy is well established, but the non-lysosomal
chaperone therapy is heterogeneous and to be investigated further for various individual
diseases. As a whole, these two-types of new molecular therapeutic approaches will
make an impact on the treatment of a wide range of pathological conditions caused
by protein misfolding, not necessarily lysosomal but also many non-lysosomal diseases
caused by gene mutations, metabolic diseases, malignancy, infectious diseases, and
aging. The concept will open a completely new aspect of protein therapy in future.
Keywords
Abbreviations:
CMA (chaperone-mediated autophagy), DGJ (1-deoxygalactonojirimycin), ER (endoplasmic reticulum), ERT (enzyme replacement therapy), α-Gal A (α-galactosidase A), β-Gal (β-galactosidase), β-Glu (β-glucosidase), Hsf1 (heat shock factor 1), Hsp (heat shock protein), 6S-NBI-DGJ (5N,6S-(N-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin), NOEV (N-octyl-4-epi-β-valienamine), NOV (N-octyl-β-valienamine), polyQ (polyglutamine), PQC (protein quality-control)To read this article in full you will need to make a payment
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Article info
Publication history
Published online: March 02, 2023
Accepted:
February 15,
2023
Received in revised form:
January 20,
2023
Received:
December 16,
2022
Publication stage
In Press Corrected ProofIdentification
Copyright
© 2023 Published by Elsevier B.V. on behalf of The Japanese Society of Child Neurology. All rights reserved.
