Drug intelligence / Profile preview

shRBM5

Development stage
Preclinical
Lead developer
Soochow University
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Parenteral
01

Overview

shRBM5 is a short hairpin RNA (shRNA) designed to silence the expression of the RNA-binding protein RBM5 (RNA Binding Motif Protein 5) via RNA interference (RNAi). Research presented at ASH 2023 indicates that RBM5 is highly expressed in acute myeloid leukemia (AML) and is essential for the survival and progression of HOXA9-driven leukemias. By targeting RBM5 mRNA, shRBM5 reduces RBM5 protein levels, which in turn impairs the transcriptional activation of the oncogenic protein HOXA9. In preclinical models, treatment with shRBM5 has been shown to inhibit AML cell growth, induce myeloid differentiation, and reduce leukemic infiltration in bone marrow and spleen, suggesting its potential as a therapeutic strategy for AML.

Other names
RBM5 shRNARBM-5 shRNARBM 5 shRNAshort hairpin RNA targeting RBM5
02

Targets

RNA binding motif protein 5 mRNA

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