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The Biological Approach Video

Biological Approach

Many diseases appear to be caused by the misregulation of protein maintenance. Such diseases of protein homeostasis, or "proteostasis," include loss-of-function diseases cystic fibrosis and gain-of-toxic-function diseases Biologial, Parkinson's, and Huntington's disease. Proteostasis is maintained by the proteostasis network, which comprises pathways that control protein synthesis, folding, The Biological Approach, aggregation, disaggregation, and degradation.

Herein, we review recent evidence supporting the principle that proteostasis is influenced both by an adjustable proteostasis network capacity and protein folding energetics, which together determine the balance between folding efficiency, misfolding, protein degradation, and aggregation.

We review how small molecules can enhance proteostasis by binding to and stabilizing specific proteins pharmacologic chaperones or by increasing the proteostasis network capacity proteostasis regulators. We propose that such therapeutic strategies, including combination therapies, represent a new approach for treating a range of diverse human maladies.

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Abstract Many diseases appear to be caused by the misregulation of protein maintenance. Publication types Research Support, N. Gov't Review.

Substances Molecular Chaperones Proteins.]