Long believed to be nonfunctional, mutant proteins that cause disease can often behave normally when refolded with the help of a pharmacological template
Many diseases are caused by mutations in a person's DNA, since these errors often change the amino acid composition of proteins encoded by genes. Scientists who study genetic diseases frequently experiment on cells that contain the mutation and produce the defective protein, which usually has altered chemical properties.
Figure 1. The Japanese art of origami can create a complex three-dimensional sculpture from a single sheet of paper through a series of precise folds and twists, as shown in this pairing of template and finished product. Likewise, a protein begins with a one-dimensional string of amino acids and, by a chaperone-assisted process of folding, becomes an intricate three-dimensional object that can perform nearly any task that the cell may require. However, mutations in a person's DNA can cause changes in the sequence of amino acids and prevent a protein from folding normally—a circumstance that often causes disease. Recent evidence from the authors indicates that many errant mutants of one receptor protein can regain normal function when folded with the help of a "pharmacoperone." The approach may hold promise for treating dozens of other genetic diseases.
Origami silverfish and template appear courtesy of Robert J. Lang
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