Protein Patched Homolog 1 mRNA (PTCH1 mRNA)

PTCH1 mRNA is the messenger RNA transcript of the Protein Patched Homolog 1 (PTCH1) gene, which encodes a critical transmembrane receptor and tumor suppressor in the Hedgehog (Hh) signaling pathway. In the absence of Hh ligands, the PTCH1 protein inhibits the activity of Smoothened (SMO), thereby preventing the activation of downstream GLI transcription factors. Mutations in the PTCH1 gene, particularly those affecting mRNA splicing or stability, lead to constitutive Hh pathway activation, which is a primary driver of basal cell carcinoma, medulloblastoma, and Gorlin syndrome. Additionally, PTCH1 mRNA is a target for endogenous microRNAs, such as miR-9 and miR-212, which can downregulate its expression and contribute to chemotherapy resistance in glioblastoma and lung cancer. While current clinical interventions primarily target the downstream SMO protein with small molecules like vismodegib, PTCH1 mRNA is being investigated as a target for novel RNA-based therapies, including mRNA replacement and splice-modulating antisense oligonucleotides. It also serves as a significant biomarker for patient stratification in cancers where Hh signaling is dysregulated.

Other names

Patched 1 mRNAPTC1 mRNAPTCH mRNANBCCS mRNAProtein patched homolog 1 transcript