Article

Integrating Machine Learning-Based Approaches into the Design of ASO Therapies

2025-01-03

Abstract excerpt

Rare diseases impose a significant burden on affected individuals, caregivers, and healthcare systems worldwide. Developing effective therapeutics for these small patient populations presents substantial challenges. Antisense oligonucleotides (ASOs) have emerged as a promising therapeutic approach that targets the underlying genetic cause of disease at the RNA level. Several ASOs have gained FDA approval for the t...

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Literature Corpus work: cf4dab64-07d0-55a3-83b1-28c0549737da
DOI: 10.20944/preprints202501.0135.v1

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