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Transforming growth factor beta 1 (TGF-β1) messenger RNA is the primary transcript responsible for the production of the TGF-β1 protein, a potent pleiotropic cytokine that acts as a master regulator of immune homeostasis, cell growth, and tissue repair [1][4]. In various pathological conditions, particularly chronic organ fibrosis and advanced-stage malignancies, TGF-β1 mRNA is significantly overexpressed, driving disease progression by promoting the epithelial-mesenchymal transition (EMT) and the excessive accumulation of extracellular matrix components like collagen [3]. Targeting the mRNA directly using RNA-based therapeutics, such as antisense oligonucleotides (ASOs) or small interfering RNAs (siRNAs), allows for the silencing of TGF-β1 expression at the pre-translational level, potentially offering more complete inhibition than protein-targeting antibodies [2]. For example, the siRNA candidate STP705 is designed to target both TGF-β1 and COX-2 mRNA to treat fibrotic skin diseases and certain cancers [2]. However, because TGF-β1 also functions as a critical tumor suppressor in early-stage tissues and maintains peripheral immune tolerance, therapeutic targeting of its mRNA requires precise delivery and dosing to avoid adverse effects such as systemic inflammation or impaired tissue regeneration [1][3].
Antisense inhibition or RNA interference (RNAi) leading to mRNA degradation or translation blockade, thereby reducing the production of the TGF-beta 1 protein.
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