Asset

  • No.

    145

  • Asset Title

    AAV Vectors for Direct Delivery of shRNA into Islet Cells to Treat Pancreatic Cancer

  • Organization

    University of Florida

  • Product Type

    Gene therapy

  • Therapeutic Area

    Oncology

  • Development Stage

    Hit To Lead or Lead Optimization

  • Technical Summary

      shRNA reduces thymidylate synthase (TS) levels, significantly decreasing the progression of pancreatic neuroendocrine tumors (PanNETs). 

    • This treatment employs an AAV-TS vector that specifically targets pancreatic islet cells. The vector contains small hairpin RNA (shRNA) molecules and releases them into identified pancreatic islet cells. 

    • This reduces thymidylate synthase (TS) levels, significantly decreasing the progression of pancreatic neuroendocrine tumors (PanNETs). TS acts as a biomarker and therapeutic target.

     • Although TS plays a central role in DNA synthesis/repair and is essential for cell proliferation, high levels of TS correlate strongly with tumorigenesis, poor therapeutic outcomes, and low overall survival rates in cancer patients. • A mouse with an hTS/Men1 (-/-) allele established a model to replicate the human disease of PanNET to test how the interfering RNA targeted the TS. 

  • Patent

    US20190256858A1

  • Publication

    Thymidylate synthase accelerates Men1-mediated pancreatic tumor progression and reduces survival. JCI insight, (2022)

  • Attachment

TOP