| 摘要 |
<p>The subject disclosure relates to strategies and methods for the discovery, development, and use of drugs and drug lead molecules that inhibit protein folding (folding inhibitors). These can consist of small organic molecules that bind nascent polypeptides selectively within cells during their synthesis on ribosomes and/or before folding of the protein is completed, and by virtue of this activity inhibit the target polypeptide from folding to its native state which is otherwise responsible for its biological activities. Methods for discovering folding inhibitors include, for example: 1) the design of specific peptide and non-peptide inhibitors, 2) the identification of suitable chemistries for the synthesis of combinatorial libraries of small organic molecules, 3) aptamers, and 4) effective screening methods. The present invention also relates to methods of enhancing the potencies of said compounds, which are expected to have extraordinary medicinal properties.</p> |
