Drug intelligence / Profile preview

miR-125b lentivirus

Development stage
Preclinical
Lead developer
University Health Network
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

miR-125b lentivirus is an experimental gene therapy construct consisting of a lentiviral vector designed to deliver and enforce the expression of microRNA-125b (miR-125b). miR-125b is a small non-coding RNA that functions as a key regulator of hematopoietic stem cell (HSC) and leukemia stem cell (LSC) maintenance by modulating the translation and stability of various target mRNAs. In preclinical studies of acute myeloid leukemia (AML), the use of this lentiviral vector to over-express miR-125b has been shown to promote the expansion of LSCs and multi-lymphoid progenitors (MLP), suggesting its role as an oncomiR that enhances self-renewal. This tool is primarily used in research settings to investigate the molecular mechanisms of leukemogenesis and stem cell biology.

Other names
miR-125b-expressing lentivirusmiR125b-expressing lentivirusmiR 125b-expressing lentiviruslentiviral miR-125b
02

Targets

Apelin (APLN) mRNATNFAIP3 (Tumor necrosis factor alpha-induced protein 3)

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