Abstract
In some instances, small molecules can restore function to proteins that are impaired by genetic mutations. There are now many examples where non-specific molecules or specific ligands can act as chemical chaperones to fold proteins or stabilize folded proteins harboring genetic mutations. In contrast a few recent examples have shown that functionally impaired proteins that are stably folded can be “functionally rescued” by appropriate small molecules. Compounds that can rescue functionally impaired proteins may provide new strategies for the treatment of genetic diseases such as rickets and resistance to thyroid hormone (RTH). In addition mutant-complementing analogs and substrates that act exclusively on mutant proteins are providing important tools for the study of complex biological systems that are controlled by molecules that have multiple cellular targets.
Current Topics in Medicinal Chemistry
Title: Ligand-receptor Engineering and its Application Towards the Complementation of Genetic Disease and Target Identification
Volume: 5 Issue: 4
Author(s): John T. Koh and John B. Biggins
Affiliation:
Abstract: In some instances, small molecules can restore function to proteins that are impaired by genetic mutations. There are now many examples where non-specific molecules or specific ligands can act as chemical chaperones to fold proteins or stabilize folded proteins harboring genetic mutations. In contrast a few recent examples have shown that functionally impaired proteins that are stably folded can be “functionally rescued” by appropriate small molecules. Compounds that can rescue functionally impaired proteins may provide new strategies for the treatment of genetic diseases such as rickets and resistance to thyroid hormone (RTH). In addition mutant-complementing analogs and substrates that act exclusively on mutant proteins are providing important tools for the study of complex biological systems that are controlled by molecules that have multiple cellular targets.
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Cite this article as:
Koh T. John and Biggins B. John, Ligand-receptor Engineering and its Application Towards the Complementation of Genetic Disease and Target Identification, Current Topics in Medicinal Chemistry 2005; 5 (4) . https://dx.doi.org/10.2174/1568026053828420
DOI https://dx.doi.org/10.2174/1568026053828420 |
Print ISSN 1568-0266 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4294 |
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