Drug intelligence / Profile preview

sgRBM5

Development stage
Preclinical
Lead developer
Soochow University
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
01

Overview

sgRBM5 refers to single-guide RNAs (sgRNAs) designed to target the RNA Binding Motif Protein 5 (RBM5) gene using CRISPR/Cas9 technology. In research presented at the ASH 2023 meeting, these sgRNAs were utilized in genome-wide CRISPR/Cas9 screens to identify RBM5 as a critical survival factor in Acute Myeloid Leukemia (AML). RBM5 is an RNA-binding protein that acts as a regulator of the oncogenic protein HOXA9. Functional validation using sgRBM5 demonstrated that the loss of RBM5 significantly impairs AML cell growth, reduces colony formation, and triggers myeloid differentiation. While currently employed as a research tool for functional genomics and target validation, RBM5 represents a novel therapeutic target for HOXA9-driven leukemias.

Other names
sgRNA against RBM5single guide RNA targeting RBM5sgRNA-RBM5sgRNA-RBM-5sgRNA-RBM 5
02

Targets

RBM5

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