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Article

Bottlebrush Polymer Conjugates for Enhanced Antisense Oligonucleotide Therapy in Myotonic Dystrophy Type 1

2025-08-08

Abstract excerpt

Oligonucleotides are a promising genetic medicine for myotonic dystrophy type 1 (DM1), the most common adult-onset muscular dystrophy. However, poor muscle distribution of nucleic acid drugs after systemic administration has hindered drug development, and no curative treatment exists. Additionally, DM1 pathology requires drug localization to the nucleus, where pathogenic mutant RNA is trapped, posing challenges af...

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Identifiers and source

Literature Corpus work
f9850ce9-6d94-5d3e-b90e-5009e221ad85
DOI
10.1101/2025.08.06.668589
Open publication

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