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Biomedicines 2018, 6(1), 9;

RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis

Department of Neurology, Nerve-Muscle Unit and ALS Center, CHU Bordeaux (Pellegrin Hospital), F-33000 Bordeaux, France
Neurocentre Magendie, Physiopathologie de la Plasticité Neuronale, University of Bordeaux, U862, F-33000 Bordeaux, France
INSERM, Neurocentre Magendie, Physiopathologie de la Plasticité Neuronale, U862, F-33000 Bordeaux, France
Author to whom correspondence should be addressed.
Received: 7 December 2017 / Revised: 7 January 2018 / Accepted: 9 January 2018 / Published: 15 January 2018
(This article belongs to the Special Issue Antisense Therapy)
Full-Text   |   PDF [260 KB, uploaded 15 January 2018]


Amyotrophic lateral sclerosis (ALS) is a fatal motor disease in adults. Its pathophysiology remains mysterious, but tremendous advances have been made with the discovery of the most frequent mutations of its more common familial form linked to the C9ORF72 gene. Although most cases are still considered sporadic, these genetic mutations have revealed the role of RNA production, processing and transport in ALS, and may be important players in all ALS forms. There are no disease-modifying treatments for adult human neurodegenerative diseases, including ALS. As in spinal muscular atrophy, RNA-targeted therapies have been proposed as potential strategies for treating this neurodegenerative disorder. Successes achieved in various animal models of ALS have proven that RNA therapies are both safe and effective. With careful consideration of the applicability of such therapies in humans, it is possible to anticipate ongoing in vivo research and clinical trial development of RNA therapies for treating ALS. View Full-Text
Keywords: antisense oligonucleotide (ASO); ASOs; therapy; RNA; ALS antisense oligonucleotide (ASO); ASOs; therapy; RNA; ALS
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Mathis, S.; Le Masson, G. RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis. Biomedicines 2018, 6, 9.

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