Special Issue "Recent Advances in Amyloid Research: Molecular Biology, Pharmacology, Pathophysiology, Diagnostic Technology, and Therapeutics"

A special issue of Biomedicines (ISSN 2227-9059).

Deadline for manuscript submissions: closed (15 January 2019)

Special Issue Editors

Guest Editor
Prof. Masahide Yazaki

Department of Biological Sciences for Intractable Neurological Diseases, Institute for Biomedical Sciences, Shinshu University, Matsumoto, Japan
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Interests: ATTR amyloidosis; diagnostic technology; biochemical features of amyloid fibrils
Guest Editor
Prof. Yoshiki Sekijima

Department of Neurology and Rheumatology, Shinshu University School of Medicine, Matsumoto, Japan
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Interests: ATTR amyloidosis; biochemical featutes of amyloid fibrils; cell biology; amyloid imaging; therapeutics

Special Issue Information

Dear Colleagues,

Amyloidosis is one of the representative misfolded protein disorders, resulting from intra or extracellular accumulation of amyloid fibrils. While amyloidosis was previously quite an intractable disease, recent basic research on the pathogenesis of several amyloidoses has gradually been able to elucidated several types of amyloidosis, which have become treatable. However, we need more detailed information on amyloidosis and its pathogenesis. Therefore, this Special Issue of Biomedicines will focus on recent advance in amyloid research, including new insights into molecular biology, pharmacology, pathophysiology, and therapeutics. In addition, as it will be more important to make a correct diagnosis in the early stages of the disease after the establishment of effective treatments, submissions regarding new diagnostic procedures are welcome.

Prof. Masahide Yazaki
Prof. Yoshiki Sekijima
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomedicines is an international peer-reviewed open access quarterly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 550 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • Pharmacology
  • Pathology
  • Pathophysiology
  • Biochemistry
  • diagnostic technology
  • new therapies
  • cell biology
  • animal models

Published Papers (1 paper)

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Review

Open AccessReview
Ultrastructure in Transthyretin Amyloidosis: From Pathophysiology to Therapeutic Insights
Biomedicines 2019, 7(1), 11; https://doi.org/10.3390/biomedicines7010011
Received: 3 January 2019 / Revised: 20 January 2019 / Accepted: 24 January 2019 / Published: 5 February 2019
Cited by 3 | PDF Full-text (6907 KB) | HTML Full-text | XML Full-text
Abstract
Transthyretin (TTR) amyloidosis is caused by systemic deposition of wild-type or variant amyloidogenic TTR (ATTRwt and ATTRv, respectively). ATTRwt amyloidosis has traditionally been termed senile systemic amyloidosis, while ATTRv amyloidosis has been called familial amyloid polyneuropathy. Although ATTRwt amyloidosis has classically been regarded [...] Read more.
Transthyretin (TTR) amyloidosis is caused by systemic deposition of wild-type or variant amyloidogenic TTR (ATTRwt and ATTRv, respectively). ATTRwt amyloidosis has traditionally been termed senile systemic amyloidosis, while ATTRv amyloidosis has been called familial amyloid polyneuropathy. Although ATTRwt amyloidosis has classically been regarded as one of the causes of cardiomyopathy occurring in the elderly population, recent developments in diagnostic techniques have significantly expanded the concept of this disease. For example, this disease is now considered an important cause of carpal tunnel syndrome in the elderly population. The phenotypes of ATTRv amyloidosis also vary depending on the mutation and age of onset. Peripheral neuropathy usually predominates in patients from the conventional endemic foci, while cardiomyopathy or oculoleptomeningeal involvement may also become major problems in other patients. Electron microscopic studies indicate that the direct impact of amyloid fibrils on surrounding tissues leads to organ damage, whereas accumulating evidence suggests that nonfibrillar TTR, such as oligomeric TTR, is toxic, inducing neurodegeneration. Microangiopathy has been suggested to act as an initial lesion, increasing the leakage of circulating TTR. Regarding treatments, the efficacy of liver transplantation has been established for ATTRv amyloidosis patients, particularly patients with early-onset amyloidosis. Recent phase III clinical trials have shown the efficacy of TTR stabilizers, such as tafamidis and diflunisal, for both ATTRwt and ATTRv amyloidosis patients. In addition, a short interfering RNA (siRNA), patisiran, and an antisense oligonucleotide (ASO), inotersen, have been shown to be effective for ATTRv amyloidosis patients. Given their ability to significantly reduce the production of both wild-type and variant TTR in the liver, these gene-silencing drugs seem to be the optimal therapeutic option for ATTR amyloidosis. Hence, the long-term efficacy and tolerability of novel therapies, particularly siRNA and ASO, must be determined to establish an appropriate treatment program. Full article
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