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Specific biomarkers for C9orf72 FTD/ALS could expedite the journey towards effective therapies

Balendra, R; Moens, TG; Isaacs, AM; (2017) Specific biomarkers for C9orf72 FTD/ALS could expedite the journey towards effective therapies. EMBO Molecular Medicine , 9 (7) , Article e201707848. 10.15252/emmm.201707848. Green open access

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Abstract

A hexanucleotide repeat expansion in the C9orf72 gene is a common genetic cause of ALS and FTD. The repeats are translated into five different dipeptide repeat proteins (DPRs). In this issue, Lehmer et al (2017) demonstrate that one of these DPRs, poly(GP), can be measured in the CSF of individuals with C9orf72 mutations. In conjunction with the findings from another recent study (Gendron et al, 2017), these DPR biomarkers may prove to be extremely valuable in the quest for effective therapies for C9FTD/ALS.

Type: Article
Title: Specific biomarkers for C9orf72 FTD/ALS could expedite the journey towards effective therapies
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.15252/emmm.201707848
Publisher version: http://doi.org/10.15252/emmm.201707848
Language: English
Additional information: © 2017 The Authors. This is an open access article under the terms of the Creative Commons Attribution 4.0 License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Neurodegenerative Diseases
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences
URI: https://discovery.ucl.ac.uk/id/eprint/1557860
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