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Ad-shTGF-beta1 is an investigational gene therapy construct consisting of a recombinant adenoviral vector engineered to deliver a short hairpin RNA (shRNA) targeting the Transforming Growth Factor beta-1 (TGF-beta1) gene. TGF-beta1 is a potent pro-fibrotic and immunosuppressive cytokine that plays a critical role in the development of organ fibrosis, the promotion of epithelial-mesenchymal transition (EMT), and the inhibition of anti-tumor immune responses. Upon transduction of target cells, the adenoviral vector facilitates the expression of shRNA, which is subsequently processed by the cellular RNA interference (RNAi) machinery into small interfering RNAs (siRNAs). these siRNAs guide the RNA-induced silencing complex (RISC) to degrade TGF-beta1 mRNA, thereby knocking down protein production. This therapeutic approach is primarily being explored for the treatment of liver cirrhosis, hypertrophic scars, and various solid tumors where TGF-beta1 overexpression contributes to disease progression.
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