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UBE3A small interfering RNA + UBE3A short hairpin RNA
- Development stage
- Preclinical
- Lead developer
- University of Ulsan
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Intracerebroventricular, Intracisterna Magna, Intracisternal, Intraparenchymal, Intrastriatal, Viral Vector-based
01
Overview
UBE3A small interfering RNA + UBE3A short hairpin RNA is an experimental combination of RNA interference (RNAi) agents designed to silence the expression of the UBE3A gene. UBE3A, also known as E6AP, is an E3 ubiquitin ligase that is frequently overexpressed in glioblastoma (GBM) and correlates with poor patient prognosis. By utilizing both siRNA and shRNA to achieve robust knockdown of UBE3A, this therapeutic approach aims to inhibit the ubiquitination and subsequent degradation of SV2A. The resulting stabilization of SV2A leads to the suppression of the MAPK signaling pathway, specifically reducing the phosphorylation of ERK and p38, which inhibits tumor cell proliferation, migration, and invasion while promoting apoptosis. This combination is currently being investigated in preclinical research for its potential as a targeted therapy for glioblastoma.
- Other names
- UBE3A siRNA + UBE3A shRNAUBE3A-targeting RNAiUBE-3A-targeting RNAiUBE 3A-targeting RNAi
02
Targets
UBE3A-ATS (UBE3A antisense transcript)
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