Pursuing new genetic treatments – Fred Hutch researchers developed new approaches to treating lung disease associated with cystic fibrosis and alpha-1 antitrypsin deficiency. Dr. Miller, who recently retired, was one of the first researchers to efficiently transfer genes into human cells, a breakthrough that is providing scientists worldwide with new gene therapy tools.
Research focused on developing more efficient viral transport vehicles, or vectors, for human gene therapy. Miller and his collaborators made significant improvements in gene transfer to blood cells and immune system cells. The ability to transfer genes into such cells is critical to achieving the long-term integration of curative genes for a variety of diseases, including genetic diseases and certain cancers.