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Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses
Misfolding and extracellular deposition of proteins is the hallmark of a heterogeneous group of conditions collectively termed protein misfolding and deposition diseases or amyloidoses. These include both localized (e.g. Alzheimer’s disease, prion diseases, type 2 diabetes mellitus) and systemic amy...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2020
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7366848/ https://www.ncbi.nlm.nih.gov/pubmed/32754033 http://dx.doi.org/10.3389/fphar.2020.01024 |
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author | Nevone, Alice Merlini, Giampaolo Nuvolone, Mario |
author_facet | Nevone, Alice Merlini, Giampaolo Nuvolone, Mario |
author_sort | Nevone, Alice |
collection | PubMed |
description | Misfolding and extracellular deposition of proteins is the hallmark of a heterogeneous group of conditions collectively termed protein misfolding and deposition diseases or amyloidoses. These include both localized (e.g. Alzheimer’s disease, prion diseases, type 2 diabetes mellitus) and systemic amyloidoses. Historically regarded as a group of maladies with limited, even inexistent, therapeutic options, some forms of systemic amyloidoses have recently witnessed a series of unparalleled therapeutic successes, positively impacting on their natural history and sometimes even on their incidence. In this review article we will revisit the most relevant of these accomplishments. Collectively, current evidence converges towards a crucial role of an early and conspicuous reduction or stabilization of the amyloid-forming protein in its native conformation. Such an approach can reduce disease incidence in at risk individuals, limit organ function deterioration, promote organ function recovery, improve quality of life and extend survival in diseased subjects. Therapeutic success achieved in these forms of systemic amyloidoses may guide the research on other protein misfolding and deposition diseases for which effective etiologic therapeutic options are still absent. |
format | Online Article Text |
id | pubmed-7366848 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-73668482020-08-03 Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses Nevone, Alice Merlini, Giampaolo Nuvolone, Mario Front Pharmacol Pharmacology Misfolding and extracellular deposition of proteins is the hallmark of a heterogeneous group of conditions collectively termed protein misfolding and deposition diseases or amyloidoses. These include both localized (e.g. Alzheimer’s disease, prion diseases, type 2 diabetes mellitus) and systemic amyloidoses. Historically regarded as a group of maladies with limited, even inexistent, therapeutic options, some forms of systemic amyloidoses have recently witnessed a series of unparalleled therapeutic successes, positively impacting on their natural history and sometimes even on their incidence. In this review article we will revisit the most relevant of these accomplishments. Collectively, current evidence converges towards a crucial role of an early and conspicuous reduction or stabilization of the amyloid-forming protein in its native conformation. Such an approach can reduce disease incidence in at risk individuals, limit organ function deterioration, promote organ function recovery, improve quality of life and extend survival in diseased subjects. Therapeutic success achieved in these forms of systemic amyloidoses may guide the research on other protein misfolding and deposition diseases for which effective etiologic therapeutic options are still absent. Frontiers Media S.A. 2020-07-10 /pmc/articles/PMC7366848/ /pubmed/32754033 http://dx.doi.org/10.3389/fphar.2020.01024 Text en Copyright © 2020 Nevone, Merlini and Nuvolone http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Pharmacology Nevone, Alice Merlini, Giampaolo Nuvolone, Mario Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title | Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title_full | Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title_fullStr | Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title_full_unstemmed | Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title_short | Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses |
title_sort | treating protein misfolding diseases: therapeutic successes against systemic amyloidoses |
topic | Pharmacology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7366848/ https://www.ncbi.nlm.nih.gov/pubmed/32754033 http://dx.doi.org/10.3389/fphar.2020.01024 |
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