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SMAD-NIS-MSCs

Development stage
Preclinical
Lead developer
Ludwig Maximilian University of Munich
Modality
Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

SMAD-NIS-MSCs are genetically engineered bone marrow-derived mesenchymal stem cells (MSCs) designed for tumor-targeted gene therapy and imaging. These cells are stably transfected with a plasmid containing the sodium iodide symporter (NIS) gene under the control of a synthetic SMAD-responsive promoter. This design leverages the high levels of transforming growth factor beta 1 (TGFB1) typically found in the tumor microenvironment; TGFB1 signaling activates the SMAD pathway, which in turn drives the expression of NIS specifically within the tumor stroma. Once expressed, NIS allows for the uptake of radionuclides such as Iodine-123 for non-invasive scintigraphic imaging of MSC homing, or Iodine-131 for targeted radiotherapy. Preclinical studies in hepatocellular carcinoma models have demonstrated significant tumor growth delay and improved survival using this approach.

Other names
SMAD-responsive NIS-expressing MSCsSMAD-NIS-MSC

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