Drug intelligence / Profile preview

shMsi2-3

Development stage
Preclinical
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Ex Vivo, Viral/lentiviral Transduction
01

Overview

shMsi2-3 is a **short hairpin RNA (shRNA) therapeutic agent** specifically designed to silence the Musashi-2 (Msi2) gene, an RNA-binding protein that is implicated in leukemogenesis and poor clinical prognosis in acute myeloid leukemia (AML). By targeting and downregulating Msi2 expression, shMsi2-3 inhibits AML cell proliferation, induces G0/G1 cell cycle arrest, and promotes apoptosis. This is mechanistically associated with decreased phosphorylation of Akt, Erk1/2, and p38 signaling pathways, resulting in suppressed survival and enhanced chemosensitivity to treatment with daunorubicin. Experimental studies with AML cell lines (Dami, HL-60) and primary AML cells demonstrate tangible in vitro and in vivo effects, including prolonged survival in xenograft mouse models. shMsi2-3 is experimental and developed as a gene silencing approach for investigational therapy in acute myeloid leukemia[1].

02

Targets

MSI2 (Musashi RNA-binding protein 2)

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