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pMETTL3 + isPEG-LPD
- Development stage
- Preclinical
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
pMETTL3 + isPEG-LPD is an experimental CRISPR/Cas9-based gene therapy system designed for the treatment of hepatocellular carcinoma (HCC). It consists of a CRISPR/Cas9 plasmid targeting the Methyltransferase-Like 3 (METTL3) gene (pMETTL3) encapsulated within a multifunctional nano-delivery system. The delivery vehicle, termed isPEG-LPD, utilizes a cationic polyethyleneimine (PEI) backbone modified with deoxycholic acid (DOCA) for hydrophobicity and lactobionic acid (LA) for active targeting of HCC cells via the asialoglycoprotein receptor. The system is further functionalized with pH-sensitive, cleavable polyethylene glycol (isPEG) to enhance circulation time and reduce toxicity. Upon reaching the acidic tumor microenvironment, the PEG layer is shed, allowing for cellular uptake and subsequent CRISPR-mediated disruption of METTL3, which inhibits tumor cell proliferation and induces apoptosis.
- Other names
- pMETTL3/isPEG-LPD
02
Targets
METTL3 (Methyltransferase-like protein 3)
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