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Splice-Switching ASO Collapses Oncogenic Circuit in Pancreatic Cancer Models

The peer-reviewed study demonstrates activity in human tumor organoids, signaling an early preclinical stage.

Overview

  • Cold Spring Harbor researchers mapped an SRSF1AURKAMYC feedback loop that drives aggressive PDAC progression.
  • SRSF1 promotes an AURKA splicing pattern that boosts AURKA, which stabilizes MYC, reinforcing SRSF1 to sustain the loop.
  • From 12 antisense oligonucleotides, a lead candidate, ASO-A, forced AURKA exon skipping, collapsed the circuit, and triggered apoptosis in tumor cells.
  • The approach reduced viability in PDAC cell models and human patient-derived organoids, offering a potential complement to KRAS-targeted therapies.
  • The findings, published in Molecular Cell, come as the Krainer lab continues optimizing the ASO before further preclinical work and any consideration of clinical testing.