Molecular Therapeutics

We are currently administrating two projects to develop new concept drugs for a variety of human diseases. One is gene therapy, and the other is to isolate therapeutically valuable biological response modifiers from herbs used in traditional oriental medicines. Although these two projects are seemingly different and distant to each other, they do share a common theme, which is to obtain therapeutic affects through the regulation of gene expression by using other genes or other chemical compound(s), in this case those that have been isolated from herbs. Furthermore, experimental techniques employed in these two projects are virtually identical.

(1) Gene medicine

Gene therapy is medical intervention based on genetic modification of living cells, either indirectly through an ex vivo approach or directly by applying the gene to the human subjects. One of the major limiting factors in this field is the lack of a gene delivery system that can efficiently and safely transfer the therapeutic gene to the body. We have recently developed improved gene delivery vehicles based on murine leukemia virus and naked DNA (plasmid) that are much superior to other existing systems. Using these tools, we have developed or are developing a variety of gene-based drugs that can be used for various life-threatening diseases. Our group played a significant role in carrying out Korea's first gene therapy clinical trial in the year 2001. Five clinical trials are planned in the US, Europe, China, and Korea over the next three years for genetic diseases, cancer and rheumatoid arthritis.

(2) Molecular herbal medicines


Another project is to isolate BRM from traditional herbs and develop them into new drugs. Although many therapeutically valuable herbs and formulations (consisting of multiple herbs) are available in Korea thanks to its rich history in the field, there has been very slow scientific progress. This is due mainly to a lack of understanding of modern science by oriental medicine physicians, underestimation of traditional herbal medicine by west-oriented molecular biologists and western medicine physicians, and a lack of cooperation between these two groups of people. We have secured a variety of herbal formulations that are unavailable to the public from the country's finest oriental medicine physicians and studied them at the molecular level as well as in the animal model and in vitro cell culture systems. A few recently obtained data include: a) water-soluble extract from an edible mushroom that highly activates transcription factor NF-kB and induces the differentiation of and proliferation of granulocyte lineage cells from the bone marrow; b) ethanol-extract from 12 different herbs that have strong anti-inflammatory and cartilage protective effects by regulating related cytokines and enzymes; c) water-soluble extract from an edible fruit at a specific growth stage that regulates Th2 response and has the potential for anti-atopic agents. As of February 2003, we are working on seven leading formulations.