Text republished from the UCLA Office of Intellectual Property & Industry Sponsored Research: https://techtransfer.universityofcalifornia.edu/NCD/20445.htmlA number of antisense oligonucleotide agents are currently in clinical trials for a wide range of diseases. Antisense technology is broadly used by the pharmaceutical industry as a tool for functional genomics and for highly specific drugs in different therapeutic areas. Antisense oligonucleotides in clinical trials are frequently found to be too inefficient to cause a sufficient amount of exon skipping to be therapeutically effective. To date, no molecule that can increase the efficiency of antisense mediated skipping has been identified.Researchers at UCLA have discovered a series of compounds that facilitate therapeutic exon skipping. The compounds were derived from FDA approved libraries or known biologically active molecule libraries. The molecules were identified via a small molecule library screen using a cell reporter assay. Some compounds have been demonstrated to increase the amount of mRNA that is skipped in the presence of antisense therapeutics.APPLICATIONSEnhancement of the therapeutic effect of antisense oligonucleotides when used as a combination treatmentIncrease in the amount of mRNA that is skipped in the presence of antisense therapeuticsADVANTAGESEnhancement of the therapeutic effect of antisense treatments that are currently too inefficient to be effectiveThe compounds were derived from FDA-approved libraries of known biologically active molecule librariesImage credit: RNA Molecule, Blausen.com staff. "Blausen gallery 2014". Wikiversity Journal of Medicine. DOI:10.15347/wjm/2014.010. ISSN 20018762
