AIMC Topic: Oligonucleotides, Antisense
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Integrating Machine Learning-Based Approaches into the Design of ASO Therapies.
Genes
Feb 2, 2025
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) ...
Mitigating off-target effects of small RNAs: conventional approaches, network theory and artificial intelligence.
British journal of pharmacology
Sep 18, 2024
Three types of highly promising small RNA therapeutics, namely, small interfering RNAs (siRNAs), microRNAs (miRNAs) and the RNA subtype of antisense oligonucleotides (ASOs), offer advantages over small-molecule drugs. These small RNAs can target any ...
Development and validation of AI/ML derived splice-switching oligonucleotides.
Molecular systems biology
Apr 25, 2024
Splice-switching oligonucleotides (SSOs) are antisense compounds that act directly on pre-mRNA to modulate alternative splicing (AS). This study demonstrates the value that artificial intelligence/machine learning (AI/ML) provides for the identificat...
Blood-brain barrier crossing biopolymer targeting c-Myc and anti-PD-1 activate primary brain lymphoma immunity: Artificial intelligence analysis.
Journal of controlled release : official journal of the Controlled Release Society
May 10, 2025
Primary Central Nervous System Lymphoma is an aggressive central nervous system neoplasm with poor response to pharmacological treatment, partially due to insufficient drug delivery across blood-brain barrier. In this study, we developed a novel ther...
eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping.
Nucleic acids research
Jul 2, 2021
Exon skipping using antisense oligonucleotides (ASOs) has recently proven to be a powerful tool for mRNA splicing modulation. Several exon-skipping ASOs have been approved to treat genetic diseases worldwide. However, a significant challenge is the d...
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