Drug intelligence / Profile preview

CBFB retroviral vector

Development stage
Preclinical
Lead developer
Washington University in St. Louis
Modality
Gene Therapies
Administration
Parenteral
01

Overview

CBFB retroviral vector is an experimental gene therapy tool designed to restore the expression of the Core Binding Factor Beta (CBFB) subunit in hematopoietic cells. CBFB is a critical component of the Core Binding Factor (CBF) transcription complex, which also includes the DNA-binding subunit RUNX1. In the context of Acute Myeloid Leukemia (AML), reduced CBFB expression is frequently observed, leading to impaired hematopoiesis and compensatory upregulation of RUNX1. This retroviral vector is used in preclinical research to demonstrate that restoring CBFB levels can normalize RUNX1 expression and potentially reverse preleukemic phenotypes, suggesting a therapeutic strategy for AML patients with CBF pathway deficiencies.

Other names
CBFB-expressing retroviral vectorretroviral CBFB expression vector
02

Targets

RUNX (Runt-related transcription factor (RUNX) family)

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