Daniel Guthrie Miller, MD, PhD

Daniel Guthrie Miller, MD, PhD


On staff since October 2003

Academic Title: Associate Professor

Research Center: Center for Immunity and Immunotherapies

"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.

Making a Difference

  • Mentoring Talented Junior Faculty

    An academic medical center and research institute, Seattle Children’s is dedicated to helping junior faculty establish research careers. Recently, several of our emerging genetics researchers have made significant contributions to their field. ... cont.


  • Kate Bellevue, WA 06.05.12

    We only saw Dr. Miller one time and got a good impression for him. He is very attentive, smart and spent a LOT of time with our family explaining all the issues/concerns that we had, offering tests that other doctors failed to mention, and answering all of our questions. He was easy to talk to. He doesn't make you feel like you are a lesser person an he is THE doctor.. like a lot of doctors of his level tend to do. He also paid attention to our kid, which was nice, most doctors tend to ignore the little ones and treat them as subjects that they are "treating".


Board Certification(s)

Clinical Genetics (MD)

Medical/Professional School

University of Washington School of Medicine, Seattle


Pediatrics, University of Washington School of Medicine, Seattle


Medical Genetics, University of Washington School of Medicine, Seattle

Clinical Interests

Gene therapy, cellular mechanisms of chromosomal translocation and deletion

Research Focus Area

Genetics and Developmental Biology


  • Miller DG, Lovell EO
    Antibiotic-induced serotonin syndrome.
    18455905 The Journal of emergency medicine , 2011 Jan. : 25-7
  • Snider L, Geng LN, Lemmers RJ, Kyba M, Ware CB, Nelson AM, Tawil R, Filippova GN, van der Maarel SM, Tapscott SJ, Miller DG
    Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene.
    21060811 PLoS genetics , 2010 Oct. 28 : e1001181
  • Lemmers RJ, van der Vliet PJ, Klooster R, Sacconi S, Camaño P, Dauwerse JG, Snider L, Straasheijm KR, van Ommen GJ, Padberg GW, Miller DG, Tapscott SJ, Tawil R, Frants RR, van der Maarel SM
    A unifying genetic model for facioscapulohumeral muscular dystrophy.
    20724583 Science (New York, N.Y.) , 2010 Sept. 24 : 1650-3
  • Petek LM, Fleckman P, Miller DG
    Efficient KRT14 targeting and functional characterization of transplanted human keratinocytes for the treatment of epidermolysis bullosa simplex.
    20571545 Molecular therapy : the journal of the American Society of Gene Therapy , 2010 Sept. : 1624-32
  • Stadler B, Ivanovska I, Mehta K, Song S, Nelson A, Tan Y, Mathieu J, Darby C, Blau CA, Ware C, Peters G, Miller DG, Shen L, Cleary MA, Ruohola-Baker H
    Characterization of microRNAs involved in embryonic stem cell states.
    20128659 Stem cells and development , 2010 July : 935-50
  • Petek LM, Russell DW, Miller DG
    Frequent endonuclease cleavage at off-target locations in vivo.
    20216527 Molecular therapy : the journal of the American Society of Gene Therapy , 2010 May : 983-6
  • Snider L, Asawachaicharn A, Tyler AE, Geng LN, Petek LM, Maves L, Miller DG, Lemmers RJ, Winokur ST, Tawil R, van der Maarel SM, Filippova GN, Tapscott SJ
    RNA transcripts, miRNA-sized fragments and proteins produced from D4Z4 units: new candidates for the pathophysiology of facioscapulohumeral dystrophy.
    19359275 Human molecular genetics , 2009 July 1 : 2414-30
  • Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS
    AAV vector integration sites in mouse hepatocellular carcinoma.
    17656716 Science (New York, N.Y.) , 2007 July 27 : 477
  • Beard BC, Dickerson D, Beebe K, Gooch C, Fletcher J, Okbinoglu T, Miller DG, Jacobs MA, Kaul R, Kiem HP, Trobridge GD
    Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells.
    17440443 Molecular therapy : the journal of the American Society of Gene Therapy , 2007 July : 1356-65
  • Beard BC, Keyser KA, Trobridge GD, Peterson LJ, Miller DG, Jacobs M, Kaul R, Kiem HP
    Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus.
    17518616 Human gene therapy , 2007 May : 423-34
  • Aker M, Tubb J, Miller DG, Stamatoyannopoulos G, Emery DW
    Integration bias of gammaretrovirus vectors following transduction and growth of primary mouse hematopoietic progenitor cells with and without selection.
    16647882 Molecular therapy : the journal of the American Society of Gene Therapy , 2006 Aug. : 226-35
  • Miller DG, Wang PR, Petek LM, Hirata RK, Sands MS, Russell DW
    Gene targeting in vivo by adeno-associated virus vectors.
    16878127 Nature biotechnology , 2006 Aug. : 1022-6
  • Trobridge GD, Miller DG, Jacobs MA, Allen JM, Kiem HP, Kaul R, Russell DW
    Foamy virus vector integration sites in normal human cells.
    16428288 Proceedings of the National Academy of Sciences of the United States of America , 2006 Jan. 31 : 1498-503
  • Miller DG, Trobridge GD, Petek LM, Jacobs MA, Kaul R, Russell DW
    Large-scale analysis of adeno-associated virus vector integration sites in normal human cells.
    16103194 Journal of virology , 2005 Sept. : 11434-42
  • Gregorevic P, Blankinship MJ, Allen JM, Crawford RW, Meuse L, Miller DG, Russell DW, Chamberlain JS
    Systemic delivery of genes to striated muscles using adeno-associated viral vectors.
    15273747 Nature medicine , 2004 Aug. : 828-34
  • Miller DG, Petek LM, Russell DW
    Adeno-associated virus vectors integrate at chromosome breakage sites.
    15208627 Nature genetics , 2004 July : 767-73
  • Hirata RK, Xu C, Dong R, Miller DG, Ferguson S, Russell DW
    Efficient PRNP gene targeting in bovine fibroblasts by adeno-associated virus vectors.
    15107244 Cloning and stem cells , 2004 : 31-6
  • Miller DG, Petek LM, Russell DW
    Human gene targeting by adeno-associated virus vectors is enhanced by DNA double-strand breaks.
    12724413 Molecular and cellular biology , 2003 May : 3550-7
  • Miller DG, Rutledge EA, Russell DW
    Chromosomal effects of adeno-associated virus vector integration.
    11799395 Nature genetics , 2002 Feb. : 147-8