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

Abstract

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 diseases & disorders > congenital hereditary genetic diseases bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > DNA expression bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification diseases & disorders bioinformatics > genomics and proteomics > genetics & nucleic acid processing bioinformatics > genomics and proteomics bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > antisense organs, tissues, organelles, cell types and functions > organs types and functions > brain diseases & disorders > cancer > cancer types > huntington's disease bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > oligonucleotide organs, tissues, organelles, cell types and functions > organs types and functions organs, tissues, organelles, cell types and functions diseases & disorders > congenital hereditary genetic diseases > spinal muscular atrophy
CSHL Authors:	Krainer, Adrian R.
Communities:	CSHL labs > Krainer lab
Depositing User:	Matthew Dunn
Date:	8 July 2019
Date Deposited:	08 Aug 2019 13:39
Last Modified:	01 Feb 2024 20:31
PMCID:	PMC7427431
Related URLs:	Publisher
URI:	https://repository.cshl.edu/id/eprint/38138

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