Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.

Publication date: Jul 08, 2019

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.

Bennett, C.F., Krainer, A.R., and Cleveland, D.W. Antisense Oligonucleotide Therapies for Neurodegenerative Diseases. 18544. 2019 Annu Rev Neurosci (42):

Concepts Keywords
Alzheimer Neurological diseases
Amyotrophic Lateral Sclerosis Spinal muscular atrophy
Antisense Drug Progression disease
Antisense Drugs Neurodegenerative diseases
Huntington Branches of biology
Intrathecal Neurological disorders
Intrathecally Spinal muscular atrophy
Neurodegenerative Diseases Senescence
Neurological Diseases Antisense therapy
Oligonucleotide Biotechnology
Oligonucleotides Gene therapy
Spinal Muscular Atrophy Nusinersen
Tolerability Degenerative disease


Type Source Name
drug DRUGBANK Spinosad
disease DOID spinal muscular atrophy
disease MESH spinal muscular atrophy
drug DRUGBANK Nusinersen
disease MESH multiple
disease DOID amyotrophic lateral sclerosis
disease MESH amyotrophic lateral sclerosis
disease MESH development
pathway BSID Neurodegenerative Diseases
disease MESH Neurodegenerative Diseases


Original Article

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