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Date: March 9, 2005


Minnesota Gene Therapy Start-up Believes Gene Therapy is Not a Dream

Discovery Genomics, Inc. (DGI), a Minnesota-based gene therapy company, has developed a unique solution that it believes will overcome current problems with gene therapy treatments and lead the way to making gene therapy a viable medical treatment.

Gene therapy is the process by which a healthy gene is delivered to cells to correct a malfunctioning gene. For gene therapy to fulfill its promise it must ensure a safe and long lasting therapeutic effect.

In most gene therapy studies, disabled virus-based vectors have been the carrier of choice for delivery of therapeutic genes to cells. Historically, these viral vectors have provided the only means of achieving long lasting therapeutic effects based on their ability to integrate into chromosomes. Unfortunately, these vectors have also presented a variety of potential problems for patients. Toxicity, immune/inflammatory responses and undesirable gene activation have contributed to serious adverse events in clinical studies of gene therapy. Many virus-based vectors have very strong promoters that can turn on cancer-causing genes in the process of inserting the therapeutic gene into a chromosome.

DGI’s proprietary Sleeping Beauty Transposon System uses pure DNA instead of viruses to carry the therapeutic genes into the cell. Additionally, we have constructed our DNA transposons so that they do not contain the same types of strong promoters implicated in the problems that have occurred in the recent clinical trials using viruses. Once in the cell our transposon places the curative genes directly into chromosomes, with therapeutic effects that last the life of the cell.

DGI has selected an orphan genetic disease, Fanconi Anemia, to validate its approach. Fanconi Anemia (FA) is the most common of the inherited anemias and leads to progressive, severe, bone marrow failure early in life. The effects of the disease are devastating and many children born with Faconi Anemia do not survive into adulthood. Therapeutic genes for this disease are known and available.

DGI’s treatment for FA involves extracting a sample of the child’s bone marrow. The cells in the bone marrow, including stem cells, are stimulated with an electric field to take up the therapeutic DNA. The cells are then returned to the child. Only a few treated stem cells may be required to cure the anemia.

DGI hopes to show that this relatively fast and inexpensive approach can greatly benefit the children suffering from FA who have no other alternative. If validated, this treatment could provide a safe and effective alternative to the viral gene therapy vectors for the treatment of other blood diseases such as hemophilia and AIDS. In the end, DGI’s Sleeping Beauty Transposon System could provide a breakthrough in providing safe and effective gene therapy.



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