Gene Transfer

The ultimate goal of investigators in the Stem Cell Institute is to develop gene therapy strategies that employ multipotent stem cells (cells capable of generating different cell types), or lineage-specific stem cells (cells capable of generating only one kind of cell type) for the treatment of human/animal disease.

Clinical gene therapy trials are being developed for treating leukemia, bone marrow diseases such as Fanconi anemia, metabolic diseases such as Hurler disease, inherited diseases such as hemophilia and muscular dystrophy, and anti-angiogenesis therapies for cancer that stop tumor-feeding blood vessels from forming.