Development of Next-Generation 'Gene Therapy Platform' That Selectively Kills Cancer Cells

KIST Team Delivers Cancer Cell-Killing Genes Directly to Tumor Tissues
Gene and Nanomaterial Fusion Enables "Recurrence-Preventing Therapy"

A team of Korean researchers has successfully developed a hybrid platform capable of effectively delivering genes that directly induce the death of cancer cells. This new platform overcomes the traditional limitations of gene therapy, such as immune system clearance and liver accumulation, and is drawing attention as a next-generation cancer immunotherapy technology by also inducing a powerful and sustained immune response.

Cover image of "Advanced Science" visualizing the tumor ablation mechanism of AAV/MnO2-PEI hybrid complexes. National Institute for Korean Medicine Development

According to the National Institute for Korean Medicine Development on October 28, a research team led by Hyojin Lee, Ph.D., at the Korea Institute of Science and Technology (KIST), under the supervision of Professor Ko Youngil at Seoul National University Hospital, has synthesized a gene that directly induces the death of cancer cells. The team coated this gene with manganese dioxide, developing a hybrid platform that effectively delivers the cancer cell-killing gene to tumor tissues without being cleared by the immune system or accumulating in the liver.

This platform has demonstrated a dual effect: it powerfully eliminates cancer cells while simultaneously enhancing the attack capabilities of immune cells.

Conventional chemotherapy faces challenges in achieving long-term therapeutic effects due to drug resistance. Similarly, gene therapies using carriers such as adeno-associated viruses (AAV) to deliver genes to specific cells have been limited by immune cell clearance or unwanted accumulation in the liver when administered in the body.

To overcome these limitations, the research team developed a novel therapeutic platform that combines viruses with inorganic nanomaterials. This next-generation composite cancer treatment strategy not only kills cancer cells but also enables the patient's own immune cells to recognize and attack the cancer.

The platform utilizes a nanomaterial containing manganese (MnO2-PEI) to protect the gene therapy agents from external threats and ensure their stable delivery to tumor tissues. Once inside the cancer cells, the nanomaterial decomposes, generating various components that further induce the destruction of cancer cells.

As a result, the study not only demonstrated effective tumor reduction but also confirmed, through animal experiments, the formation of long-term immune memory that prevents recurrence, allowing immune cells to defend against future cancer development.

Hyojin Lee, Ph.D., Korea Institute of Science and Technology (KIST)

Dr. Hyojin Lee stated, "This study is significant in that it presents a multifunctional hybrid therapeutic strategy that overcomes the limitations of existing gene therapies by combining cancer cell ablation and immune activation. This platform has the potential to be expanded as a multifunctional carrier that can simultaneously deliver various functional compounds, such as gene-editing materials, in addition to therapeutic genes for cancer. We plan to thoroughly verify its clinical applicability through further research."

This research was supported by the Ministry of Health and Welfare's 'Research-driven Hospital Fostering Hanmi Innovation Performance Creation R&D Project' and the Ministry of Science and ICT's 'Nano and Material Technology Development Project - Material Global Young Connect Project.' It was selected as the cover article for the international journal "Advanced Science" in August.

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