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Article

Oligonucleotide therapeutics in neurodegenerative diseases

21 Mar 2018

Abstract excerpt

Therapeutics that directly target RNAs are promising for a broad spectrum of disorders, including the neurodegenerative diseases. This is exemplified by the FDA approval of Nusinersen, an antisense oligonucleotide (ASO) therapeutic for spinal muscular atrophy (SMA). RNA targeting therapeutics are currently under development for amyotrophic lateral sclerosis (ALS), Huntington's disease (HD), and spinocerebellar...

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PMID: 29560813
DOI: 10.1080/15476286.2018.1454812

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