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Systemic Amyloidosis: a Contemporary Overview

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Abstract

Amyloidosis constitutes a large spectrum of diseases characterized by an extracellular deposition of a fibrillar aggregate, generating insoluble and toxic amasses that may be deposited in tissues in bundles with an abnormal cross-β-sheet conformation, known as amyloid. Amyloid may lead to a cell damage and an impairment of organ function. Several different proteins are recognized as able to produce amyloid fibrils with a different tissue tropism related to the molecular structure. The deposition of amyloid may occur as a consequence of the presence of an abnormal protein, caused by high plasma levels of a normal protein, or as a result of the aging process along with some environmental factors. Although amyloidosis is rare, amyloid deposits play a role in several conditions as degenerative diseases. Thus, the development of antiamyloid curative treatments may be a rational approach to treat neurodegenerative conditions like Alzheimer’s disease in the future. Nowadays, novel treatment options are currently refined through controlled trials, as new drug targets and different therapeutic approaches have been identified and validated through modern advances in basic research. Fibril formation stabilizers, proteasome inhibitors, and immunotherapy revealed promising results in improving the outcomes of patients with systemic amyloidosis, and these novel algorithms will be effectively combined with current treatments based on chemotherapeutic regimens. The aim of this review is to provide an update on diagnosis and treatment for systemic amyloidosis.

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Authors and Affiliations

  1. Department of Sense Organs, Sapienza University of Rome, Viale del Policlinico 155, 00186, Rome, Italy

    Vittorio D’Aguanno, Massimo Ralli, Marco Artico, Francesca Yoshie Russo & Antonio Greco

  2. Department of Otolaryngology, University San Giovanni di Dio e Ruggi d’Aragona, Salerno, Italy

    Alfonso Scarpa

  3. Institute of Cell Biology and Neurobiology, IBCN-CNR, Rome, Italy

    Marco Fiore, Paola Tirassa & Cinzia Severini

  4. Department of Oral and Maxillofacial Sciences, Sapienza University of Rome, Rome, Italy

    Marco de Vincentiis

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  1. Vittorio D’Aguanno
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D’Aguanno, V., Ralli, M., Artico, M. et al. Systemic Amyloidosis: a Contemporary Overview. Clinic Rev Allerg Immunol 59, 304–322 (2020). https://doi.org/10.1007/s12016-019-08759-4

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