Target intelligence / Profile preview

Transforming growth factor beta-3 mRNA (TGFB3 mRNA) (TGFB3 mRNA)

Target
TGFB3 mRNA
Molecular classification
Nucleic acid, Messenger RNA
01

Overview

Transforming growth factor beta-3 (TGF-beta3) mRNA is the transcript of the TGFB3 gene, encoding a cytokine that belongs to the transforming growth factor beta superfamily [1.4.1, 1.4.2]. It plays a critical role in embryonic development, particularly in the fusion of the palate, and is essential for scarless wound healing and chondrogenesis [1.4.1, 1.4.3]. In pathological states like glioblastoma, high expression of TGF-beta3 mRNA is associated with increased tumor invasiveness and poor patient survival, acting as a key regulator of the TGF-beta signaling pathway [1.3.1, 1.3.2]. Unlike the TGF-beta1 and TGF-beta2 isoforms, TGF-beta3 has distinct structural properties that allow for specific therapeutic targeting using RNA-based technologies [1.3.1, 1.5.3]. Drugs such as antisense oligonucleotides (ASOs) and siRNAs are designed to selectively bind to TGF-beta3 mRNA, triggering its degradation and subsequently reducing the levels of the active protein [1.2.1, 1.3.2]. This isoform-specific approach is intended to mitigate the systemic toxicities, including cardiotoxicity and inflammation, that have hindered the clinical success of pan-TGF-beta inhibitors [1.5.1, 1.5.3]. Current research focuses on the preclinical and clinical evaluation of these ASOs for treating aggressive cancers and fibrotic conditions like systemic sclerosis [1.2.3, 1.5.3]. Monitoring TGF-beta3 mRNA levels and downstream signaling markers like SMAD2 phosphorylation serves as a vital strategy for assessing treatment efficacy and patient selection [1.3.1, 1.3.2].

Other names
TGFB3TGF-beta3ARVDARVD1RNHFTGFB3 mRNA
02

Mechanism of action

Antisense-mediated mRNA degradation and RNA interference leading to reduced protein translation.

03

Biological functions

Cell differentiationEmbryogenesisWound healingImmune responseChondrogenesis
04

Disease associations

CancerFibrosisCongenital malformationCardiovascular disease
05

Safety considerations

Off-target inhibition of other TGF-beta isoformsPotential for systemic adverse effects on tissue homeostasisChallenges in targeted delivery to the central nervous system
06

Interacting drugs

ISTH2020

1 more in the full profile.

07

Biomarkers

TGFB3 mRNA expression levelsSMAD2 phosphorylationPlasminogen activator inhibitor-1 (PAI-1) levels

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