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Antisense Oligonucleotide Therapy for Neurodevelopmental Disorders

1 Jan 2021

Abstract excerpt

Antisense oligonucleotides (ASOs) are short oligonucleotides that can modify gene expression and mRNA splicing in the nervous system. The FDA has approved ASOs for treatment of ten genetic disorders, with many applications currently in the pipeline. We describe the molecular mechanisms of ASO treatment for four neurodevelopmental and neuromuscular disorders. The ASO nusinersen is a general treatment for mutations...

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PMID: 34412058
DOI: 10.1159/000517686

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