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Gene Therapy for ALS-A Perspective. | LitMetric

Gene Therapy for ALS-A Perspective.

Int J Mol Sci

Sorbonne Université, Inserm UMRS 974, Centre of Research in Myology (CRM), Institut de Myologie, GH Pitié Salpêtrière, 75013 Paris, France.

Published: September 2019

AI Article Synopsis

Article Abstract

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 or 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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6771059PMC
http://dx.doi.org/10.3390/ijms20184388DOI Listing

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