Drug intelligence / Profile preview

siRNA (PAX6)

Development stage
Preclinical
Lead developer
Rush University Medical Center
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
Intraperitoneal
01

Overview

siRNA (PAX6) refers to small interfering RNA molecules designed to silence the expression of the PAX6 (paired box 6) gene. PAX6 is a transcription factor involved in embryonic development that has been implicated in the progression of various cancers, including glioblastoma multiforme (GBM) and other high-grade gliomas. By utilizing the RNA interference (RNAi) pathway, these siRNA molecules target and promote the degradation of PAX6 mRNA, thereby reducing PAX6 protein levels. Research conducted at institutions like Rush University Medical Center has explored the use of PAX6-targeting siRNAs (such as those provided in TriFECTA kits by Integrated DNA Technologies) to inhibit cell proliferation and progression in primary human glioma cultures.

Brand names
TriFECTA
Other names
PAX6 siRNAPAX-6 siRNAPAX 6 siRNAsmall interfering RNA targeting PAX6
02

Targets

PAX6

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