Targeting tumor EC-restricted alternative splicing isoforms with ASO


Claudia Ghigna



Institute of Molecular Genetics Luigi Luca Cavalli-Sforza National Research Council (IGM-CNR)



The overarching goal of Task 6.3.4 is the development of molecules and strategies to target alternative splicing events involved in heterogenous diseases, syndromes, and multi-systemic conditions currently lacking curative options. In light of the fact that a vigorous neo-vasculature development is characteristic of aggressive primary tumors and metastases and that Tumor Endothelial Cells (TECs) are readily accessible to therapeutic agents, targeted delivery of bio-active agents to the tumoral neo-vasculature is a particularly promising anticancer therapeutic modality. In the past the group of Dr. Ghigna identified Alternative Splicing (AS) variants of cell surface molecules restricted to TECs and with relevant roles for tumor angiogenesis. Our main interest is to use antisense oligonucleotides (ASO) targeting TEC-restricted AS variants to block neo-angiogenesis and consequently tumor progression.

Relevant Publications

  1. Di Matteo A, Belloni E, et al., Vermi W, Ghigna C. Alternative Splicing Changes Promoted by NOVA2 Upregulation in Endothelial Cells and Relevance for Gastric Cancer. Int J Mol Sci. 2023; 24: 8102

  2. Pradella D, et al., Eichmann A, Mehlen P, Ghigna C. A ligand-insensitive UNC5B splicing isoform regulates angiogenesis by promoting apoptosis. Nat Commun. 2021;12: 4872


Related Projects

PI: Claudia Coronnello