Protein Misfolding, Amyloid Formation, and Human Disease: A Summary of Progress Over the Last Decade

Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This phenomenon is associated with a range of increasingly common human disorders, including Alzheimer and Parkinson diseases, type II diabetes, a...

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Vydáno v:Annual review of biochemistry Ročník 86; s. 27
Hlavní autoři: Chiti, Fabrizio, Dobson, Christopher M
Médium: Journal Article
Jazyk:angličtina
Vydáno: United States 20.06.2017
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ISSN:1545-4509, 1545-4509
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Abstract Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This phenomenon is associated with a range of increasingly common human disorders, including Alzheimer and Parkinson diseases, type II diabetes, and a number of systemic amyloidoses. In this review, we describe this field of science with particular reference to the advances that have been made over the last decade in our understanding of its fundamental nature and consequences. We list the proteins that are known to be deposited as amyloid or other types of aggregates in human tissues and the disorders with which they are associated, as well as the proteins that exploit the amyloid motif to play specific functional roles in humans. In addition, we summarize the genetic factors that have provided insight into the mechanisms of disease onset. We describe recent advances in our knowledge of the structures of amyloid fibrils and their oligomeric precursors and of the mechanisms by which they are formed and proliferate to generate cellular dysfunction. We show evidence that a complex proteostasis network actively combats protein aggregation and that such an efficient system can fail in some circumstances and give rise to disease. Finally, we anticipate the development of novel therapeutic strategies with which to prevent or treat these highly debilitating and currently incurable conditions.
AbstractList Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This phenomenon is associated with a range of increasingly common human disorders, including Alzheimer and Parkinson diseases, type II diabetes, and a number of systemic amyloidoses. In this review, we describe this field of science with particular reference to the advances that have been made over the last decade in our understanding of its fundamental nature and consequences. We list the proteins that are known to be deposited as amyloid or other types of aggregates in human tissues and the disorders with which they are associated, as well as the proteins that exploit the amyloid motif to play specific functional roles in humans. In addition, we summarize the genetic factors that have provided insight into the mechanisms of disease onset. We describe recent advances in our knowledge of the structures of amyloid fibrils and their oligomeric precursors and of the mechanisms by which they are formed and proliferate to generate cellular dysfunction. We show evidence that a complex proteostasis network actively combats protein aggregation and that such an efficient system can fail in some circumstances and give rise to disease. Finally, we anticipate the development of novel therapeutic strategies with which to prevent or treat these highly debilitating and currently incurable conditions.
Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This phenomenon is associated with a range of increasingly common human disorders, including Alzheimer and Parkinson diseases, type II diabetes, and a number of systemic amyloidoses. In this review, we describe this field of science with particular reference to the advances that have been made over the last decade in our understanding of its fundamental nature and consequences. We list the proteins that are known to be deposited as amyloid or other types of aggregates in human tissues and the disorders with which they are associated, as well as the proteins that exploit the amyloid motif to play specific functional roles in humans. In addition, we summarize the genetic factors that have provided insight into the mechanisms of disease onset. We describe recent advances in our knowledge of the structures of amyloid fibrils and their oligomeric precursors and of the mechanisms by which they are formed and proliferate to generate cellular dysfunction. We show evidence that a complex proteostasis network actively combats protein aggregation and that such an efficient system can fail in some circumstances and give rise to disease. Finally, we anticipate the development of novel therapeutic strategies with which to prevent or treat these highly debilitating and currently incurable conditions.Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This phenomenon is associated with a range of increasingly common human disorders, including Alzheimer and Parkinson diseases, type II diabetes, and a number of systemic amyloidoses. In this review, we describe this field of science with particular reference to the advances that have been made over the last decade in our understanding of its fundamental nature and consequences. We list the proteins that are known to be deposited as amyloid or other types of aggregates in human tissues and the disorders with which they are associated, as well as the proteins that exploit the amyloid motif to play specific functional roles in humans. In addition, we summarize the genetic factors that have provided insight into the mechanisms of disease onset. We describe recent advances in our knowledge of the structures of amyloid fibrils and their oligomeric precursors and of the mechanisms by which they are formed and proliferate to generate cellular dysfunction. We show evidence that a complex proteostasis network actively combats protein aggregation and that such an efficient system can fail in some circumstances and give rise to disease. Finally, we anticipate the development of novel therapeutic strategies with which to prevent or treat these highly debilitating and currently incurable conditions.
Author Chiti, Fabrizio
Dobson, Christopher M
Author_xml – sequence: 1
  givenname: Fabrizio
  surname: Chiti
  fullname: Chiti, Fabrizio
  email: fabrizio.chiti@unifi.it
  organization: Department of Experimental and Clinical Biomedical Sciences "Mario Serio," Section of Biochemistry, Università di Firenze, 50134 Firenze, Italy; email: fabrizio.chiti@unifi.it
– sequence: 2
  givenname: Christopher M
  surname: Dobson
  fullname: Dobson, Christopher M
  email: cmd44@cam.ac.uk
  organization: Department of Chemistry, Centre for Misfolding Diseases, University of Cambridge, Cambridge CB2 1EW, United Kingdom; email: cmd44@cam.ac.uk
BackLink https://www.ncbi.nlm.nih.gov/pubmed/28498720$$D View this record in MEDLINE/PubMed
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Keywords amyloidosis
quality control
protein aggregation
protein homeostasis
conformational diseases
chaperones
functional amyloid
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PublicationTitle Annual review of biochemistry
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Snippet Peptides and proteins have been found to possess an inherent tendency to convert from their native functional states into intractable amyloid aggregates. This...
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SubjectTerms Alzheimer Disease - drug therapy
Alzheimer Disease - history
Alzheimer Disease - metabolism
Alzheimer Disease - pathology
Amyloid - chemistry
Amyloid - genetics
Amyloid - metabolism
Amyloidosis - drug therapy
Amyloidosis - history
Amyloidosis - metabolism
Amyloidosis - pathology
Diabetes Mellitus, Type 2 - drug therapy
Diabetes Mellitus, Type 2 - history
Diabetes Mellitus, Type 2 - metabolism
Diabetes Mellitus, Type 2 - pathology
Drugs, Investigational
Gene Expression Regulation
History, 21st Century
Humans
Immunoglobulin Light-chain Amyloidosis
Molecular Chaperones - genetics
Molecular Chaperones - metabolism
Molecular Targeted Therapy
Parkinson Disease - drug therapy
Parkinson Disease - history
Parkinson Disease - metabolism
Parkinson Disease - pathology
Protein Aggregation, Pathological - history
Protein Aggregation, Pathological - metabolism
Protein Aggregation, Pathological - pathology
Protein Aggregation, Pathological - prevention & control
Protein Conformation
Protein Folding
Proteostasis Deficiencies - drug therapy
Proteostasis Deficiencies - history
Proteostasis Deficiencies - metabolism
Proteostasis Deficiencies - pathology
Proteostasis Deficiencies - prevention & control
Title Protein Misfolding, Amyloid Formation, and Human Disease: A Summary of Progress Over the Last Decade
URI https://www.ncbi.nlm.nih.gov/pubmed/28498720
https://www.proquest.com/docview/1899114466
Volume 86
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