Target intelligence / Profile preview

Musashi RNA binding protein 2 (MSI2)

Target
MSI2
Molecular classification
RNA-binding protein, Translational regulator, Other (Musashi family)
01

Overview

Musashi RNA binding protein 2 is a post-transcriptional regulator expressed predominantly in stem and progenitor cells across neural, hematopoietic, and other tissues[1][4][6]. The protein possesses two RNA-recognition motifs (RRM1 and RRM2) that preferentially bind UAG motifs in the 3'-UTRs of target mRNAs, influencing their stability and translation efficiency[2][3]. MSI2 is crucial for sustaining stem cell “stemness,” cell fate decisions, and regulating lineage commitment, especially in blood and neuronal development. Overexpression and dysregulation of MSI2 are common and linked to poor clinical outcomes in many hematologic and solid cancers, driving cell proliferation, migration, metastasis, and resistance to chemotherapy[4][5][8][10]. Small molecules such as palmatine and Ro 08–2750 have recently been described, selectively inhibiting MSI2 activity, thus holding promise for directed cancer therapy[8][10]. Safety concerns are primarily those typical of stem cell regulators—therapeutic targeting requires balancing antitumor effect with preservation of normal tissue renewal[6][8].

Other names
Musashi-2RNA-binding protein Musashi homolog 2MSI2
02

Mechanism of action

Antagonism of MSI2-mediated RNA binding, resulting in decreased translation of oncogenic mRNAs and increased cellular differentiation/apoptosis[10].\nDirect binding to MSI2, preventing its interaction with 3′-UTRs of specific mRNAs[8].

03

Biological functions

Stem cell maintenanceCell fate determinationRegulation of asymmetric divisionPost-transcriptional modulation of mRNA translationPromotion of cell proliferationRestriction of cell migrationRegulation of lineage bias
04

Disease associations

Cancer (hematologic malignancies, colorectal, pancreatic, glioblastoma, breast, ovarian, other solid tumors)Other (developmental disorders, though primarily studied in cancer)
05

Safety considerations

Therapeutic targeting could disrupt stem cell functions and hematopoietic homeostasis[6].Potential off-target effects due to MSI2’s role in normal tissue renewal[8].
06

Interacting drugs

Palmatine

1 more in the full profile.

07

Biomarkers

MSI2 expression is associated with poor prognosis and increased tumor aggressiveness in various cancers; potential biomarker for patient selection and efficacy monitoring[5][8].

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