Drug intelligence / Profile preview

RCAS H3.3K27M-P2A-PDGFB

Development stage
Preclinical
Lead developer
University of Minnesota
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Parenteral
01

Overview

RCAS H3.3K27M-P2A-PDGFB is a replication-competent avian sarcoma-leukosis virus (RCAS) vector designed to co-express the histone H3.3 K27M mutation and the oncogenic driver Platelet-Derived Growth Factor Subunit B (PDGFB). This construct utilizes a self-cleaving P2A peptide to ensure stoichiometric expression of both proteins from a single transcript. It is primarily used in the RCAS/N-TVA mouse model system to study the pathogenesis of pediatric high-grade gliomas (pHGG), including diffuse intrinsic pontine glioma (DIPG). By delivering these genetic elements to Nestin-expressing neural stem cells in TVA-transgenic mice, researchers can induce tumors that recapitulate the epigenetic and histological features of human H3K27M-mutant gliomas, facilitating the study of histone modifications and the development of potential therapeutic strategies.

02

Targets

PDGFRα/β (Platelet-derived growth factor receptor alpha/beta heterodimer)TVA (Tumor virus A receptor)EED (Embryonic ectoderm development protein)PDGFRB (Platelet-derived growth factor receptor beta)

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