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TFE3 siRNA

Development stage: Preclinical
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intratumoral

Overview

TFE3 siRNA is a small interfering RNA (siRNA) designed to silence the expression of the Transcription Factor E3 (TFE3). In the context of TSC1-mutant bladder cancer, TFE3 undergoes constitutive nuclear localization and drives a transcriptional program associated with mTORC1 activation and lysosomal biogenesis. Experimental studies demonstrate that siRNA-mediated knockdown of TFE3 significantly reduces cell growth and viability in TSC1-null bladder cancer models, identifying TFE3 as a potential targetable dependency for this molecular subset of tumors. This approach aims to inhibit the aberrant transcriptional activity of TFE3 that contributes to tumor development and maintenance.

Other names: Transcription factor E3 siRNAsiTFE3siTFE-3siTFE 3

Targets

Innate immune RNA sensorsAGO2 (Argonaute RISC catalytic component 2)TFE3 (Transcription factor E3)

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