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siRNA (C19orf47-AKT2 targeting)

Development stage: Preclinical
Lead developer: The First Affiliated Hospital of Shantou University Medical College
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

Overview

siRNA (C19orf47-AKT2 targeting) is an experimental small interfering RNA designed to silence the expression of the C19orf47-AKT2 chimeric RNA. This chimera, generated through the cis-splicing of adjacent genes (C19orf47 and AKT2), is specifically detected in glioma tissues and is associated with poor prognosis in glioblastoma (GBM) patients. Research indicates that the C19orf47-AKT2 fusion likely functions as a non-coding RNA rather than a protein-coding entity. In preclinical studies using patient-derived GBM primary cells, siRNA-mediated knockdown of this chimeric RNA significantly inhibited cell proliferation, identifying it as a potential therapeutic target for the treatment of aggressive gliomas.

Other names: C19orf47-AKT2 siRNAC-19orf47-AKT2 siRNAC 19orf47-AKT2 siRNAsiRNA targeting C19orf47-AKT2

Targets

AKT2 (Rac-beta serine/threonine-protein kinase)C19orf47-AKT2 (C19orf47 exon 9–AKT2 exon 2 chimeric RNA)

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