Drug intelligence / Profile preview

TERT siRNA

Development stage
Preclinical
Lead developer
University of California, San Francisco
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
Intravenous, Intratumoral
01

Overview

TERT siRNA is a small interfering RNA (siRNA) designed to target and silence the expression of the telomerase reverse transcriptase (TERT) gene. TERT is the catalytic subunit of the telomerase enzyme, which is responsible for maintaining telomere length and is frequently upregulated in various cancers, including over 80% of glioblastomas (GBM), often due to mutations in the TERT promoter. By utilizing the RNA interference (RNAi) pathway, TERT siRNA facilitates the degradation of TERT mRNA, thereby inhibiting telomerase activity and potentially limiting the proliferative capacity of cancer cells. In research settings, such as those conducted at the University of California, San Francisco, TERT siRNA is employed to study the metabolic consequences of TERT inhibition, identifying biomarkers like lactate and glutathione (GSH) through magnetic resonance spectroscopy (MRS) to monitor therapeutic efficacy in glioblastoma models.

Other names
telomerase reverse transcriptase siRNAsiTERT
02

Targets

TERT (Telomerase)

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