Drug intelligence / Profile preview

SP1 shRNA

Development stage
Preclinical
Lead developer
Sun Yat-sen University
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

SP1 shRNA is a short hairpin RNA (shRNA) designed to silence the expression of the transcription factor SP1 (Specificity Protein 1). In the context of acute myeloid leukemia (AML), SP1 has been identified as a key driver of leukemogenesis by forming nuclear condensates through liquid-liquid phase separation (LLPS). These condensates act as functional hubs at super-enhancer regions to drive the transcription of oncogenic targets, most notably HNRNPUL1. By mediating the degradation of SP1 mRNA, SP1 shRNA disrupts these transcriptional programs, leading to impaired AML cell viability, reduced stemness, and attenuated disease progression in preclinical models. While primarily used as a research tool for target validation, it represents a therapeutic strategy for disrupting super-enhancer-driven oncogene transcription in high-risk and relapsed pediatric AML.

Other names
SP1-targeting shRNASP-1-targeting shRNASP 1-targeting shRNAshSP1shSP-1shSP 1Sp1 shRNASp-1 shRNASp 1 shRNA
02

Targets

SP1 (Transcription factor Sp1)

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