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Antisense Drugs Make Sense for Neurological Diseases

9 Oct 2020

Abstract excerpt

The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A new class of drugs-antisense oligonucleotides (ASOs)-show promise as a therapeutic platform for treating neurological diseases. ASOs are designed to bind to the RNAs either by promoting degradation of the targeted RNA or by elevating expression by RNA...

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PMID: 33035446
DOI: 10.1146/annurev-pharmtox-010919-023738

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