Emergent Properties of Proteostasis in Managing Cystic Fibrosis
Emergent Properties of Proteostasis in Managing Cystic Fibrosis
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DOI：
10.1101/cshperspect.a004499
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发表时间：
2011-02-01
影响因子：
7.2
通讯作者：
Hutt, Darren M.
中科院分区：
文献类型：
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作者：
Balch, William E.;Roth, Daniela M.;Hutt, Darren M.
囊性纤维化 (CF) 是蛋白质稳态或蛋白质稳态网络 (PN) 对多膜跨越蛋白囊性纤维化电导跨膜调节因子 (CFTR) 识别缺陷的结果 (Hutt 和 Balch (2010)。与许多引发错误折叠疾病的变异蛋白一样，突变 CFTR 具有复杂的折叠和膜运输行程，由 PN 管理以维持蛋白质组平衡，但这种平衡被破坏许多研究人员正在研究决定 CFTR 在健康和疾病中的折叠和功能的生物学途径，为开始理解和治疗性解决 PN 在疾病发作及其在衰老过程中的进展中的作用提供了独特的机会，我们讨论了通过治疗管理 PN 的新兴特性来控制 CFTR 折叠生物学的能量可能提供显着的临床益处。
Cystic fibrosis (CF) is a consequence of defective recognition of the multimembrane spanning protein cystic fibrosis conductance transmembrane regulator (CFTR) by the protein homeostasis or proteostasis network (PN) (Hutt and Balch (2010). Like many variant proteins triggering misfolding diseases, mutant CFTR has a complex folding and membrane trafficking itinerary that is managed by the PN to maintain proteome balance and this balance is disrupted in human disease. The biological pathways dictating the folding and function of CFTR in health and disease are being studied by numerous investigators, providing a unique opportunity to begin to understand and therapeutically address the role of the PN in disease onset, and its progression during aging. We discuss the general concept that therapeutic management of the emergent properties of the PN to control the energetics of CFTR folding biology may provide significant clinical benefit.