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Gene Therapy for ALS—A Perspective

Sorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2019, 20(18), 4388;
Received: 11 July 2019 / Revised: 30 August 2019 / Accepted: 3 September 2019 / Published: 6 September 2019
(This article belongs to the Special Issue Amyotrophic Lateral Sclerosis: Highlights of Its Complexity)
Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure. Recent advances in gene therapy open a new perspective to treat this disorder—particularly for the characterized genetic forms. Gene therapy approaches, involving the delivery of antisense oligonucleotides into the central nervous system (CNS) are being tested in clinical trials for patients with mutations in SOD1 or C9orf72 genes. Viral vectors can be used to deliver therapeutic sequences to stably transduce motor neurons in the CNS. Vectors derived from adeno-associated virus (AAV), can efficiently target genes and have been tested in several pre-clinical settings with promising outcomes. Recently, the Food and Drug Administration (FDA) approved Zolgensma, an AAV-mediated treatment for another MND—the infant form of spinal muscular atrophy. Given the accelerated progress in gene therapy, it is potentially a promising avenue to develop an efficient and safe cure for ALS. View Full-Text
Keywords: Gene therapy; antisense oligonucleotides; RNA interference; lentiviral vectors; AAV Gene therapy; antisense oligonucleotides; RNA interference; lentiviral vectors; AAV
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Cappella, M.; Ciotti, C.; Cohen-Tannoudji, M.; Biferi, M.G. Gene Therapy for ALS—A Perspective. Int. J. Mol. Sci. 2019, 20, 4388.

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