GLI family zinc finger 1 mRNA (GLI1 mRNA) (GLI1 mRNA)

GLI family zinc finger 1 (GLI1) mRNA is the messenger RNA transcript that encodes the GLI1 protein, a terminal effector and transcription factor in the Hedgehog (Hh) signaling pathway [NCBI Gene, 2024]. GLI1 is unique among the GLI family as it primarily functions as a transcriptional activator, driving the expression of genes critical for cell cycle progression, apoptosis resistance, and stemness [Rimkus et al., 2016]. Aberrant overexpression of GLI1 mRNA is a hallmark of various malignancies, often resulting from ligand-dependent Hh activation or mutations in pathway components like PTCH1 or SMO [Amakye et al., 2013]. Because GLI1 acts downstream of the Smoothened receptor, targeting its mRNA provides a therapeutic advantage in overcoming resistance to conventional SMO inhibitors [Rimkus et al., 2016]. Therapeutic strategies such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are designed to bind specifically to GLI1 mRNA, leading to its degradation and the subsequent silencing of the Hh signaling output in tumor cells [ClinicalTrials.gov, 2024]. Clinical and preclinical studies have explored agents like ISIS 183750 to evaluate the safety and efficacy of this transcript-targeted approach in solid tumors [ClinicalTrials.gov, 2024].

Other names

Glioma-associated oncogene homolog 1 mRNAGLI1 transcriptZinc finger protein GLI1 mRNA

Antisense inhibition or RNA interference (RNAi) targeting the GLI1 transcript to prevent translation and induce mRNA degradation, thereby reducing GLI1 protein levels and downstream Hedgehog pathway signaling [Rimkus et al., 2016].