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AUMskip
- Development stage
- Preclinical
- Lead developer
- AUM BioTech
- Modality
- Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- In Vitro (laboratory Use), Potentially Intravenous Or Subcutaneous (for Future Clinical/therapeutic Use; Current Primary Application Is Research)
01
Overview
AUMskip is a specialized antisense oligonucleotide (ASO) product developed by AUM Biotech for exon skipping applications. These self-delivering ASOs are designed to bind pre-mRNA and redirect splicing by causing specific exons to be skipped during mRNA processing. This approach can alter protein coding sequences or restore reading frames in cases of mutations, making it useful for research and potential therapeutic applications in genetic diseases and cancer. Unlike some other ASOs, AUMskip acts as a steric-blocking agent that modulates RNA splicing without eliciting RNase H-mediated cleavage of the RNA. The technology has been used in studies such as targeting NOVA1-regulated alternative splicing of hTERT in non-small cell lung cancer to shift telomerase transcript variants and reduce tumor growth[6].
- Brand names
- AUMskip
- Other names
- AUM skipAUM skip oligossdASOself-delivering ASO for exon skipping
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