Article

Quantifying and Mitigating Motor Phenotypes Induced by Antisense Oligonucleotides in the Central Nervous System

2021-02-15

Abstract excerpt

<h4>ABSTRACT</h4> Antisense oligonucleotides (ASOs) are emerging as a promising class of therapeutics for neurological diseases. When injected directly into the cerebrospinal fluid, ASOs distribute broadly across brain regions and exert long-lasting therapeutic effects. However, many phosphorothioate (PS)-modified gapmer ASOs show transient motor phenotypes when injected into the cerebrospinal fluid, ranging from...

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Literature Corpus work: 75ddd32a-d698-57d9-ab9d-7a15e99b9b99
DOI: 10.1101/2021.02.14.431096

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