A Multifunctional Aptamer Decorated Lipid Nanoparticles for the Delivery of EpCAM-targeted CRISPR/Cas9 Plasmid for Efficacious In Vivo Tumor Regression
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View abstract on PubMed
Summary
This summary is machine-generated.A novel aptamer-decorated lipid nanoparticle system efficiently delivers CRISPR/Cas9 plasmid to knockout EpCAM expression in cancer cells. This targeted gene therapy approach shows promise for anti-cancer applications.
Area Of Science
- Biotechnology
- Molecular Biology
- Cancer Research
Background
- Epithelial cell adhesion molecule (EpCAM) is overexpressed in many cancers, promoting tumor progression by regulating oncogenes.
- EpCAM's role in tumorigenesis makes it a significant target for novel anti-cancer therapies.
Purpose Of The Study
- To develop and evaluate an aptamer-decorated lipid nanoparticle system for targeted delivery of CRISPR/Cas9 to knockout EpCAM expression.
- To assess the efficacy of this system in vitro and in vivo for cancer treatment.
Main Methods
- Construction of an all-in-one CRISPR/Cas9 plasmid targeting the EpCAM gene.
- Development of synthetic, pH-responsive, aptamer-decorated lipid nanoparticles for targeted delivery.
- In vitro testing on various EpCAM-overexpressing cancer cell lines.
- In vivo validation in non-immunocompromised mouse tumor models.
Main Results
- The aptamer-decorated lipid nanoparticles selectively targeted EpCAM-overexpressing cells.
- Efficient delivery of the CRISPR/Cas9 plasmid led to the knockout of EpCAM expression.
- The system demonstrated pH-responsive properties and efficacy across different cancer cell lines.
- Successful in vivo validation in mouse tumor models confirmed therapeutic potential.
Conclusions
- The developed aptamer-decorated lipid nanoparticle system is an effective tool for delivering EpCAM-targeted CRISPR/Cas9 plasmids.
- This targeted gene editing approach holds significant promise for developing novel anti-cancer therapies.
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