Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

Bennett, C. F., Krainer, A. R., Cleveland, D. W. (July 2019) Antisense Oligonucleotide Therapies for Neurodegenerative Diseases. Annu Rev Neurosci, 42. pp. 385-406. ISSN 0147-006x

DOI: 10.1146/annurev-neuro-070918-050501


Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.

Item Type: Paper
Subjects: bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > antisense
diseases & disorders > cancer > cancer types > huntington's disease
diseases & disorders > congenital hereditary genetic diseases > spinal muscular atrophy
CSHL Authors:
Communities: CSHL labs > Krainer lab
Depositing User: Matthew Dunn
Date: 8 July 2019
Date Deposited: 08 Aug 2019 13:39
Last Modified: 01 Dec 2020 15:03
PMCID: PMC7427431
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