Stem Cell Biology

With the explosion of information about human genes and their function in the last few years, it has become clear that virtually every disease has a genetic component. Current medications interact with the proteins produced by genes, but researchers are now uncovering the potential of correcting the faulty genes themselves. In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. A carrier molecule called a vector is often used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to remove pathogenic characteristics and carry normal human DNA.

Initially, the Department focused on various aspects of gene therapy research, such as understanding basic virology, efficient gene delivery into cells, and incorporation of these genes into the genome. Ideally, a viral vector is delivered to target cells such as the patient's liver or lung cells. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. The therapeutic gene is incorporated into the cell's genome and produces a functional protein product, which restores the target cell to a normal state and potentially cures the disease.

The Department has since broadened its scope to integrate gene therapy and stem cell biology. In addition to targeting diseased tissues directly, like the liver, the Department is also studying how to deliver genes to stem cells, specifically hematopoietic stem cells (developmentally immature cells that form blood). This approach produces functioning gene product in all the cells of a specific tissue, such as all blood cells.

This combination of gene therapy and stem cell biology provides a powerful approach to new, future therapeutics. Eventually, researchers would like to selectively collect stem cells from a patient, use gene therapy in the laboratory to correct faulty genes in the stem cells, and then transplant the treated stem cells back into the patient to treat diseases like sickle cell anemia or immune deficiencies. By using a patient's own stem cells to treat disease, scientists will be able to avoid immune rejection of foreign tissues, a continuing problem in transplant biology.