Related Experiment Video
Updated: Feb 20, 2026

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?
Published on: June 13, 2014
Aptamer-targeted delivery of Bcl-xL shRNA using alkyl modified PAMAM dendrimers into lung cancer cells
Sara Ayatollahi1, Zahra Salmasi2, Maryam Hashemi2
1Pharmaceutical Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
Abstract:
RNAi-based gene therapy has been recently considered as a promising approach against cancer. Targeted delivery of drug, gene or therapeutic RNAi-based systems to tumor cells is one of the important issues in order to reduce side effects on normal cells. Several strategies have been developed to improve the safety and selectivity of cancer treatments including antibodies, peptides and recently aptamers with various attractive characteristics including higher target specificity, affinity and reduced toxicity. Here we described a novel targeted delivery platform comprising modified PAMAM with 10-bromodecanoic acid (10C) and 10C-PEG for improvement of transfection efficiency, AS1411 aptamer for targeting nucleolin ligand on target cancer cells and shRNA plasmid for specific knockdown of Bcl-xL protein. Modified vector could significantly improve the transfection efficiency even after covalent or non-covalent aptamer binding compared to the non-targeted vector in A549 cells. The results of gene silencing and apoptosis assay indicated that our targeted shRNA delivery system could efficiently down-regulate the Bcl-xL expression up to 25% and induce 14% late apoptosis in target cancer cells with strong cell selectivity. This study proposed a novel targeted non-viral system for shRNA-mediated gene-silencing in cancer cells.
Insights
This study presents a novel targeted delivery system for cancer gene therapy using modified PAMAM nanoparticles and an AS1411 aptamer. The system effectively delivers shRNA to cancer cells, reducing Bcl-xL expression and inducing apoptosis with high selectivity.
Area of Science:
- Biotechnology
- Nanomedicine
- Cancer Therapeutics
Background:
- RNA interference (RNAi)-based gene therapy offers a promising strategy for cancer treatment.
- Targeted delivery of therapeutic agents to tumor cells is crucial for enhancing efficacy and minimizing side effects.
- Aptamers offer high specificity and affinity for targeted delivery applications in cancer therapy.
Purpose of the Study:
- To develop a novel targeted non-viral delivery system for shRNA-mediated gene silencing in cancer cells.
- To enhance transfection efficiency and target cancer cells specifically using modified PAMAM nanoparticles and an AS1411 aptamer.
- To evaluate the efficacy of the targeted system in down-regulating Bcl-xL protein expression and inducing apoptosis in cancer cells.
Main Methods:
- Development of a targeted delivery platform using modified polyamidoamine (PAMAM) dendrimers with 10-bromodecanoic acid (10C) and 10C-PEG.
- Conjugation of AS1411 aptamer for targeting nucleolin on cancer cells and incorporation of shRNA plasmid for Bcl-xL knockdown.
- Evaluation of transfection efficiency in A549 cells and assessment of gene silencing and apoptosis induction via gene silencing and apoptosis assays.
Main Results:
- The modified vector demonstrated significantly improved transfection efficiency compared to non-targeted vectors in A549 cells.
- The targeted shRNA delivery system effectively down-regulated Bcl-xL expression by up to 25%.
- The system induced 14% late apoptosis in target cancer cells, exhibiting strong cell selectivity.
Conclusions:
- A novel targeted non-viral delivery system for shRNA-mediated gene silencing in cancer cells was successfully developed.
- The modified PAMAM-based vector with AS1411 aptamer enhances transfection efficiency and provides targeted delivery to cancer cells.
- This targeted system shows potential for effective and selective cancer gene therapy by down-regulating Bcl-xL and inducing apoptosis.
Related Concept Videos
Targeted Cancer Therapies
There are several types of targeted therapies against...
Site-Targeted Drug Delivery Systems: Polymeric Carriers

