‘A patient’s father who is a retired Boeing engineer likens my work to his in that we both bring intelligent people together to tackle big problems. He calls the solutions ‘planned inventions.’ Whether it’s gene therapy or a new breed of aircraft, it’s something that hasn’t previously existed. But when we saw we needed it, we brought together the right people to make it a reality.’ Daniel G. Miller, MD, PhD, is an Associate professor at Seattle Children’s Hospital and the University of Washington School of Medicine. Miller’s Laboratory is located in the Institute for Stem Cell and Regenerative Medicine at the University of Washington’s South Lake Union Research facility. He is interested in the molecular pathophysiology of Facioscapulohumeral Muscular Dystrophy and collaborates with a group of Scientists located around the world to perform studies that will determine the molecular events that cause muscle weakness in this dominantly inherited adult-onset disease. Miller also uses gene therapy strategies to study and treat human genetic diseases. He constructs vectors based on retroviruses that deliver an entire copy of the gene to patient cells for “gene replacement” therapies, and vectors based on adeno-associated virus (AAV) that pair with chromosomal sequences facilitating “gene repair” by homologous recombination. Using these techniques, he is able to modify primary human cell lines to repair of specific mutations that cause human diseases, or generate mutations similar to those causing inherited disease in patients to study the cellular effects of such mutations. Miller sees patients with genetic conditions in the Medical Genetics Clinic. Seattle Children’s provides healthcare without regard to race, color, religion (creed), sex, gender identity or expression, sexual orientation, national origin (ancestry) or disability. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho. Source.