CDUPRT-IFNb-engineered mesenchymal stem cells

CDUPRT-IFNb-engineered mesenchymal stem cells (MSCs) are an advanced cell-based gene therapy designed to provide targeted anti-tumor activity while minimizing systemic toxicity. These MSCs are genetically modified, typically using non-viral methods, to co-express a fusion protein of **cytosine deaminase (CD)** and **uracil phosphoribosyl transferase (UPRT)**, along with **Interferon-beta (IFNb)**. Leveraging the natural tumor-homing ability of MSCs, the cells migrate to the tumor microenvironment. Once there, the CD enzyme converts the systemically administered non-toxic prodrug 5-fluorocytosine (5-FC) into the cytotoxic agent 5-fluorouracil (5-FU), while UPRT further enhances the conversion of 5-FU into active metabolites that inhibit **Thymidylate synthase** and incorporate into **DNA** and **RNA**, leading to tumor cell death. The addition of IFN-beta provides a dual therapeutic effect by exerting direct anti-proliferative activity and stimulating the immune system through the **IFNAR** and the **cGAS-STING** pathway. This multi-transgene approach is primarily investigated for the treatment of difficult-to-reach or metastatic cancers such as peritoneal carcinomatosis, glioblastoma, and hepatocellular carcinoma.

Other names

CDUPRT-IFNb-armed MSCsCD/UPRT-IFN-beta-engineered mesenchymal stem cellsMesenchymal stem cells expressing cytosine deaminase, uracil phosphoribosyltransferase, and interferon-beta