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PA2G4 shRNA

Development stage
Preclinical
Lead developer
Nationwide Children's Hospital
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

PA2G4 shRNA is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of the PA2G4 (Proliferation-associated protein 2G4) gene. PA2G4, also known as EBP1 (ErbB3-binding protein 1), is a multifunctional protein that plays a critical role in cell growth and survival; its overexpression is significantly correlated with poor progression-free and overall survival in pediatric diffuse gliomas. The therapeutic approach utilizes lentiviral particles to deliver short hairpin RNA (shRNA) sequences into tumor cells, leading to a stable knockdown of PA2G4 protein levels. Preclinical research has demonstrated that this knockdown (approximately 95% reduction) significantly inhibits cell proliferation in pediatric glioblastoma cell lines such as SF188 and KNS42, suggesting that PA2G4 is a viable therapeutic target for high-grade pediatric brain tumors.

Other names
PA2G4-KDPA-2G4-KDPA 2G4-KDshRNA-mediated knockdown of PA2G4
02

Targets

EBP1 (Proliferation-associated protein 2G4)

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