Talking Biotech with Dr. Kevin Folta Targeting RNA with Therapeutics - Dr. Matthew Disney
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Oct 5, 2025 Dr. Matthew Disney, an expert in small-molecule therapeutics at the UF Scripps Institute, dives into the revolutionary potential of RNA as a therapeutic target. He explores its pathogenic roles in diseases like ALS and myotonic dystrophy, and discusses innovative strategies to design small molecules that target structured RNA. Disney also highlights how RIBOTACs can recruit RNA-degrading enzymes, and shares insights on targeting viral genomes, including strategies for inhibiting SARS-CoV-2, showcasing the promising future of RNA therapeutics in combating various diseases.
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Early RNA Drug Success Story
- Streptomycin was the first modern clinical trial drug and it targeted bacterial ribosomal RNA.
- That antibiotic cured patients of tuberculosis and famously treated Bob Dole in early trials.
RNA Is A Vast Therapeutic Space
- The human genome reveals cells are 'bags of RNA' with many regulatory and noncoding RNAs.
- Targeting RNA could potentially treat almost any disease by modulating those RNA functions.
Pathogenic Repeat RNAs Drive Disease
- Many severe diseases, like the most common cause of ALS, are driven by toxic RNA repeat expansions.
- Silencing or removing those RNAs would directly cure the disease mechanism.