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Amyotrophic lateral sclerosis – The tools of the trait

Identifieur interne : 002E55 ( Main/Corpus ); précédent : 002E54; suivant : 002E56

Amyotrophic lateral sclerosis – The tools of the trait

Auteurs : Carsten W. Lederer ; Niovi Santama

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RBID : ISTEX:2F15259809724AF8FC51AE652AD1E80CCEFA92F7

English descriptors

Abstract

The aim of this review is to analyze how our knowledge on the etiology, pathology, and treatment of amyotrophic lateral sclerosis (ALS) has profited from the application of biotechnology tools for the identification of disease markers, the development of animal disease models, and the design of innovative therapeutics. In humans, ALS‐specific clinical, genetic or protein biomarkers, or panels of biomarkers stemming from genomics and proteomics analyses can be critical for early diagnosis, monitoring of disease progression, drug validation in clinical trials, and identification of therapeutic targets for subsequent drug development. At the same time, animal models representing a number of human superoxide dismutase 1 mutations, intermediate‐filament disorganization or axonal‐transport defects have been invaluable in unraveling aspects of the pathophysiology of the disease; in each case, these only represent a small proportion of all ALS patients. Preclinical and clinical trials, although at present heavily concentrating on pharmacological approaches, are embracing the emerging alternative strategies of stem‐cell and gene therapy. In combination with a further subcategorization of patients and the development of corresponding model systems for functional analyses, they will significantly influence the already changing face of ALS therapy.

Url:
DOI: 10.1002/biot.200600247

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ISTEX:2F15259809724AF8FC51AE652AD1E80CCEFA92F7

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<detail type="volume">
<caption>vol.</caption>
<number>2</number>
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<detail type="issue">
<caption>no.</caption>
<number>5</number>
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<identifier type="DOI">10.1002/biot.200600247</identifier>
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<accessCondition type="use and reproduction" contentType="copyright">Copyright © 2007 WILEY‐VCH Verlag GmbH & Co. KGaA, Weinheim</accessCondition>
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