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ube2e3 shrna
- Development stage
- Preclinical
- Lead developer
- Hadassah Medical Center
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
UBE2E3 shRNA is a short hairpin RNA therapeutic candidate designed to silence the expression of the ubiquitin-conjugating enzyme E2 E3 (UBE2E3). Research presented at ASH 2024 identifies UBE2E3 as a critical regulator of leukemia stem cell (LSC) survival in acute myeloid leukemia (AML). UBE2E3 is imported into the nucleus by importin 11 (IPO11) and interacts with the non-canonical polycomb repression complex 1.1 (PRC1.1), including core components such as KDM2B, BCOR, RING1, and PCGF1. By positively regulating PRC1.1 activity, UBE2E3 maintains the transcriptomic signature of LSCs and promotes the expression of target genes like PKM, MAP3K6, and ERCC1. Silencing UBE2E3 via shRNA in AML cell lines (such as OCI-AML2, NB4, and K562) has been shown to reduce cell growth, viability, and clonogenic growth while shifting the transcriptomic profile toward a more differentiated state, suggesting that targeting UBE2E3 may be a viable strategy for eradicating leukemia initiating cells.
- Other names
- shRNA targeting UBE2E3UBE2E3 short hairpin RNAUBE-2E3 short hairpin RNAUBE 2E3 short hairpin RNA
02
Targets
UBE2E3 (Ubiquitin-conjugating enzyme E2 E3)
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