C Frank Bennett, Adrian R Krainer, Don W Cleveland
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引用次数: 184
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.
期刊介绍：
The Annual Review of Neuroscience is a well-established and comprehensive journal in the field of neuroscience, with a rich history and a commitment to open access and scholarly communication. The journal has been in publication since 1978, providing a long-standing source of authoritative reviews in neuroscience.
The Annual Review of Neuroscience encompasses a wide range of topics within neuroscience, including but not limited to: Molecular and cellular neuroscience, Neurogenetics, Developmental neuroscience, Neural plasticity and repair, Systems neuroscience, Cognitive neuroscience, Behavioral neuroscience, Neurobiology of disease. Occasionally, the journal also features reviews on the history of neuroscience and ethical considerations within the field.
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