Article ; Online: Chaperone therapy for lysosomal and non-lysosomal protein misfolding diseases.
2023 Volume 45, Issue 5, Page(s) 251–259
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 ... ...
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. |
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MeSH term(s) | Humans ; Molecular Chaperones/metabolism ; Proteostasis Deficiencies/genetics ; Proteostasis Deficiencies/therapy ; Proteostasis Deficiencies/metabolism ; Mutation ; Lysosomes/metabolism |
Chemical Substances | Molecular Chaperones |
Language | English |
Publishing date | 2023-03-02 |
Publishing country | Netherlands |
Document type | Journal Article ; Review |
ZDB-ID | 604822-5 |
ISSN | 1872-7131 ; 0387-7604 |
ISSN (online) | 1872-7131 |
ISSN | 0387-7604 |
DOI | 10.1016/j.braindev.2023.02.004 |
Database | MEDical Literature Analysis and Retrieval System OnLINE |
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