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Translational Neuroscience pp 99–109Cite as

Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis

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Abstract

Antisense oligonucleotides (ASO) are short DNA-like chemicals that bind to RNA by Watson–Crick base pairing and modulate function of the RNA. These chemicals do not cross the blood brain barrier, but may be delivered directly to the cerebral spinal fluid (CSF) to achieve widespread distribution throughout the brain and spinal cord. ASO have been used to target genes associated with familial amyotrophic lateral sclerosis (ALS), such as SOD1 and C9orf72 as well as miRNAs. A Phase I trial for SOD1-targeting ASO showed excellent safety and important pharmacodynamics. ASO are a promising therapeutic approach for ALS.

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Author information

Authors and Affiliations

  1. Department of Neurology, Washington University, 660 S. Euclid, Box 8111, St. Louis, MO, 63110, USA

    Wade K. Self BA & Timothy M. Miller MD, PhD

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  1. Wade K. Self BA
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  2. Timothy M. Miller MD, PhD
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Correspondence to Timothy M. Miller MD, PhD .

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Editors and Affiliations

  1. UnivofCA-SanDiego,VeteransAdmintnMedCntr, Translational Neuroscience Institute, La Jolla, California, USA

    Mark H. Tuszynski

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Self, W.K., Miller, T.M. (2016). Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis. In: Tuszynski, M. (eds) Translational Neuroscience. Springer, Boston, MA. https://doi.org/10.1007/978-1-4899-7654-3_6

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