Article

Endosomal Escape and Nuclear Localization: Critical Barriers for Antisense Therapeutics

2024-10-25

Abstract excerpt

Antisense oligonucleotides (ASOs) have emerged as a promising therapeutic approach for a wide variety of diseases, offering the potential to modulate gene expression with a high degree of specificity. These small, synthetic nucleic acid-like molecules provide unique advantages over traditional pharmacological treatments, including the ability to target previously "undruggable" genes. Despite this promise, several...

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Identifiers and source

Literature Corpus work: 75789217-c6fd-56ab-bede-bc3811bb3494
DOI: 10.20944/preprints202410.2061.v1

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