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pMETTL3 + sPEG-LPD
- Development stage
- Preclinical
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Biodegradable Polymers → Polymer-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
pMETTL3 + sPEG-LPD is an experimental multifunctional nano-targeted delivery system designed for the treatment of hepatocellular carcinoma (HCC). It utilizes CRISPR/Cas9 gene-editing technology to target and disrupt the expression of Methyltransferase-Like 3 (METTL3), a key m6A methyltransferase that promotes HCC cell proliferation and tumor growth. The delivery vehicle (sPEG-LPD) is composed of a cationic polyethyleneimine (PEI) backbone modified with deoxycholic acid (DOCA) for improved DNA binding and lysosomal escape, and lactobionic acid (LA) for active targeting of asialoglycoprotein receptors (ASGPR) overexpressed on HCC cells. The complex is further coated with pH-sensitive, cleavable polyethylene glycol (sPEG) to enhance systemic stability, prolong circulation time, and reduce off-target toxicity. Upon reaching the acidic tumor microenvironment, the sPEG layer is cleaved, facilitating cellular uptake and subsequent gene editing to induce apoptosis and inhibit tumor progression.
- Other names
- LA-DOCA-PEI/pMETTL3/sPEGCRISPR/Cas9-METTL3 nano-delivery system
02
Targets
ASGPR (Asialoglycoprotein Receptor 1)METTL3 (Methyltransferase-like protein 3)
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