The UW’s leadership in human genome sequencing technology and mapping has spawned new opportunities for its already active programs in gene therapy and blood stem cell biology. Genomics has opened the door for gene therapy, according to Dr. George Stamatoyannopoulos, professor of medicine, head of the Division of Medical Genetics and director of the Markey Molecular Medicine Center.

Stamatoyannopoulos said the field is still in its infancy. The first clinical trials were performed little more than a decade ago, and the therapeutic potential has been barely tapped. Some of the most prominent targets for gene therapy are blood diseases, such as sickle cell anemia, beta thalassemia, adenosine deaminase deficiency, and cancers. However, researchers are also investigating gene transfer into other tissues to treat patients with damaged hearts, Alzheimer’s, and liver disease, for example.

Adult stem cell research is moving forward on several fronts at the UW. A grant from the National Institute of Diabetes and Digestive and Kidney Diseases will enable researchers, with Stamatoyannopoulos as principal investigator, to study the molecular biology of blood stem cells and stem cell genes that play a key role in cell differentiation and proliferation. Investigators have already cloned and sequenced several thousand genes expressed in stem cells. The grant will enable researchers to develop strategies to analyze the genes’ functions. Related projects will examine genes affecting white blood cells and those implicated in some leukemias that run in families.

Another large program project grant supports the application of gene therapy in clinical trials. One study involves treating sickle cell anemia and beta thalassemia by introducing therapeutic genes into blood-forming stem cells and expressing these genes in red blood cells. In other studies, researchers are examining the prevention of graft-versus-host disease, which sometimes occurs in stem cell transplant recipients. There are also gene therapy trials for cystic fibrosis patients.

The UW is also committed to applying these new areas of research to actual clinical trials, and to that end has created a clinical Gene and Cell Therapy Core Laboratory. Housed at UW Medical Center’s General Clinical Research Center and funded in part by the National Center for Research Resources, this $3 million facility will enable gene and cell therapy researchers to move potentially therapeutic strategies from their labs into a clinical setting. One potential use, for example, involves removing immune system cells from HIV-positive patients, genetically modifying these cells to make them resistant to HIV infection, and growing the cells to expand cytotoxic T cells. These cells are infused back into the patients with the hope of improving their ability to fight the HIV infection.

Central to the function of this facility is the ability to manipulate patient tissue samples outside the body under extremely stringent standards of safety and purity demanded by the current Good Manufacturing Practice regulations.