Overview
The mission of the Wellstone MDSRC of Seattle is to advance new therapies for muscular dystrophies. Our laboratories conduct basic research to deepen the understanding of disease mechanisms and pathologies, identifying novel therapeutic targets. That basic research then serves as the basis of translational and preclinical work, bridging the basic science with the clinic. Our clinicians collect natural histories and biologic samples, bridging back to the laboratories by providing necessary resources to interrogate disease processes. Meanwhile, they also assemble the infrastructure and develop the relationships with patients and their families that will be necessary to conduct clinical trials. Together, our collaborative team of MDs and PhDs position our MDSRC to both develop and test novel treatments for muscular dystrophies and related diseases.
Get in touch
Potential donors, researchers, and members of the public are welcome to contact the Seattle MDSRC.
Contact us ▸
Learn more
Learn more about the NIH Muscular Dystrophy Coordinating Committee (MDCC) and the MD-CARE Act.
MDCC website ▸
Donate
MDSRC Members
DIRECTOR
Jeffrey S. Chamberlain, Ph.D.
McCaw Endowed Chair in Muscular Dystrophy, University of Washington
Professor, Department of Neurology, University of Washington
Professor, Department of Biochemistry, University of Washington
Professor of Medicine, Division of Medical Genetics, University of Washington
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The main focus of our research is on the muscular dystrophies with two major goals: to develop a better understanding of the molecular basis of the pathophysiology of the diseases, and to develop gene and cell therapies that will correct and treat the muscular dystrophies. Major targets for therapy include Duchenne muscular dystrophy and LGMD2I. Currently, the laboratory includes about 15 scientists conducting cutting edge studies that we hope to translate into clinical trials in the near future. We are working with some of the best doctors and scientists at Children's Hospital, Fred Hutchinson Cancer Research Center, University of Rochester, and Harborview Medical Center to most effectively tackle the challenge ahead: to find a treatment for muscular dystrophy.
Laboratory
Dr. Chamberlain's laboratory is located on the main campus of the University of Washington Medical Center.
Laboratory website▸
Contact Information
jsc5@uw.edu | (206) 616-6645
1959 NE Pacific St.
HSB, Room K243
Seattle, Washington 98195-7720
Co-Director
Stephen J. Tapscott, M.D., Ph.D.
Professor, Department of Neurology, University of Washington
Member, Human Biology Division, Fred Hutchinson Cancer Research Center
Member, Clinical Research Division, Fred Hutchinson Cancer Research Center
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The Tapscott Lab studies gene transcription and expression in normal development and disease, with an additional emphasis on rhabdomysarcomas (cancers with characteristics of skeletal muscle) and human muscular dystrophies. Other research areas include gene and cell therapies for muscular dystrophy, and the biology of triplet repeats and their associated diseases.
Laboratory
Dr. Tapscott's laboratory is located at the Fred Hutchinson Cancer Research Center. Laboratory website▸
Contact Information
stapscot@fhcrc.org | (206) 667-4499
1100 Fairview Avenue N.
P.O. Box 19024
C3-168
Seattle, Washington 98109-1024
Member
Joel R. Chamberlain, Ph.D.
Research Assistant Professor, Department of Medical Genetics, University of Washington
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The Chamberlain Lab studies the disease mechanisms of FSHD, developing novel disease models and utilizing patient samples. We are also developing an AAV gene-therapy based treatment for FSHD using RNAi. Other projects in the lab include developing therapies for DM1.
Laboratory
Dr. Chamberlain's laboratory is located on the main campus of the University of Washington Medical Center.
Contact Information
jrcham@uw.edu | (206) 616-6645
1959 NE Pacific St.
HSB, Room K243
Seattle, Washington 98195-7720
Member
Martin K. (Casey) Childers, D.O., Ph.D.
Professor, Department of Rehabilitation Medicine, University of Washington
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Gene therapy for myotubular myopathy, a congenital muscle disease, that causes severe and fatal weakness of the breathing muscles is a focus of the Childers lab. Our international research group (led by Dr. Anna Buj-Bello, Genethon, Evry, France and Dr. Alan Beggs, Harvard, Boston) is working to develop this new gene therapy technology to translate findings in animals to treat human patients. The Childers lab also uses a technique to “reprogram” small samples of patient blood or urine into pluripotent stem cells that can be tested in a petri dish without harm to the patient. Using these powerful techniques our hope is to find and develop new therapy for inherited muscle diseases.
Laboratory & Clinic
Dr. Childers' laboratory is located on the South Lake Union campus of the University of Washington Medical Center. His clinic is at the Rehabilitation Medicine Clinic at the University of Washington Medical Center.
Laboratory website▸
Contact Information
mkc8@uw.edu | (206) 897-5761
850 Republican St.
Room S421
Seattle, Washington 98109
Member
Seth D. Friedman, Ph.D.
Medical Physicist, Department of Radiology, Seattle Children's Hospital
Research Associate Professor, Department of Radiology, University of Washington
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Dr. Friedman runs the Radiology Clinical Research Imaging Core, a campus-wide resource under the Center for Clinical and Translational Research. His recent work seeks to coordinate novel uses of imaging focused on a wide-range of neurological and systemic disease conditions (e.g., growth hormone releasing hormone treatment with aging, mitochondrial disease, thalassemia, facioscapulohumeral muscular dystrophy (FSHD), pyridoxine dependent epilepsy). Additionally, his work examines inhibitory neurotransmitter changes in pediatric concussion and a multi-site study of FSHD integrating MRI and blood/tissue-based measures.
Clinic
Dr. Friedman's clinic is located on the main campus of Seattle Children's Hospital.
Contact Information
sfriedmn@uw.edu
MA.7.220 - Radiology
4800 Sand Point Way NE
Seattle, Washington 98105
Member
Stephen D. Hauschka, Ph.D.
Professor, Department of Biochemistry, University of Washington
Adjunct Professor, Department of Zoology, University of Washington
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Research in our group addresses basic questions of skeletal and cardiac muscle biology: (1.) How do mesodermal cells become determined to enter the skeletal and cardiac muscle cell lineages? (2.) How are skeletal and cardiac muscle genes activated during development? (3.) How are quantitative modulations in gene expression controlled with respect to fast and slow skeletal muscle fiber types and cardiac muscles; and how are these controls modulated by the unique physiology of different anatomical muscles? (4.) How can the understanding of muscle gene regulation be developed into effective strategies for muscle gene therapy? (5.) What mechanisms regulate the mitogenic responsiveness of satellite cells during skeletal muscle regeneration? (6.) Can skeletal muscle cells and stem cells be modified so as to provide functionally beneficial replacements for injured skeletal and cardiac muscle?
Laboratory
Dr. Hauschka's laboratory is located on the main campus of the University of Washington Medical Center.
Laboratory website▸
Contact Information
haus@uw.edu | (206) 616-6998
1959 NE Pacific St.
HSB, Room J541
Seattle, Washington 98195-7720
Member
Guy Odom, Ph.D.
Research Associate Professor, Neurology, University of Washington
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Our research focuses on neuromuscular disease with an emphasis on the muscular dystrophies (MD’s). As an example, gene mutations that affect production of the protein dystrophin are the cause of Duchenne muscular dystrophy (DMD) the most common lethally inherited disorder of children. A primary research interest in the Odom lab is to develop a more thorough understanding of the inherent muscle biology occurring during MD disease progression. Additionally, to identify contributory protein-protein interactions, biomarkers, & the like as potential therapeutic targets. As such, the labs’ research generally involves developing or improving genetic-based therapies. To that end, approaches often utilized include viral vector technology, high-resolution microscopy, omics technologies, incorporation of machine learning-based modeling, applying non-invasive imaging techniques & evaluating striated muscle contractile performance in dystrophic animal models.
Laboratory
Dr. Odom's laboratory is located on the main campus of the University of Washington Medical Center.
Laboratory website▸
Contact Information
godom@uw.edu | (206) 221-5412
1959 NE Pacific St.
HSB Box# 357720
Seattle, Washington 98195-7720
Member
Stanley R. Riddell, M.D.
Scientific Director, Immunotherapy Integrated Research Center, Fred Hutchinson Cancer Research Center
Member, Program in Immunology, Fred Hutchinson Cancer Research Center
Professor, School of Medicine, University of Washington
Attending Physician, Seattle Cancer Care Alliance
Scientific Co-Founder, Juno Therapeutics
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Dr. Riddell’s research focuses on 1) understanding the roles of distinct T cell subsets in protective immunity to pathogens and tumors and 2) the development and clinical application of adoptive T cell therapies for viral diseases and cancers, including with genetically modified antigen-specific T cells. His early studies demonstrated the potential to augment T cell immunity by the adoptive transfer of antigen-specific T cells and uncovered mechanisms by which virally-infected cells evade immune recognition. These findings provided insights into similar mechanisms that tumors use to escape T cell immunity and that might be therapeutically targeted to improve patient outcomes.
Dr. Riddell’s lab has developed critical techniques for isolation, expansion, genetic modification and reinfusion of therapeutic T cells, and for monitoring patient safety, T cell persistence, migration and function post-infusion. These include state-of-the-art ways to identify the tumor cell “antigens” recognized by T cells. Many are now broadly employed in adoptive immunotherapies for cancer that use natural targeting molecules called T cell receptors (TCRs) or use synthetic TCR- and antibody-related molecules known as chimeric antigen receptors (CARs).
Laboratory
Dr. Riddell's laboratory is located at the Fred Hutchinson Cancer Research Center.
Laboratory website▸
Contact Information
sriddell@fredhutch.org | (206) 667-7791
1100 Fairview Avenue N.
P.O. Box 19024
D3-100
Seattle, Washington 98109-1024
Member
Dennis W. W. Shaw, M.D.
Division Chief, Magnetic Resonance Imaging, Seattle Children's Hospital
Division Chief, Vascular/Interventional Imaging, Seattle Children's Hospital
Professor, Center for Clinical and Translational Research, Seattle Children's Hospital
Staff Radiologist, Department of Radiology, Seattle Children's Hospital
Professor, Department of Pediatric Radiology, University of Washington
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My work involves using imaging technology for diagnosis and in the guidance of minimally invasive interventions. It is very gratifying to employ these techniques to improve outcomes and decrease the discomfort of treatment for childhood diseases. I also value highly the interactions I have with the wonderful group of excellent pediatric specialists here at Children's. I help them apply and interpret the increasingly sophisticated imaging available to their clinical work with children. We work together in the research arena to increase our understanding, and ultimately enhance our treatment, of diseases that afflict children.
Clinic
Dr. Shaw's clinic is located on the main campus of Seattle Children's Hospital.
Laboratory website▸
Contact Information
ddshaw@uw.edu | (206) 987-2134
MA.7.220 - Radiology
4800 Sand Point Way NE
Seattle, Washington 98105
Member
Rabi Tawil, M.D.
Fields Endowed Professorship in Neurology, Department of Neurology, University of Rochester
Director, The Richard Fields Center for FSHD Research, University of Rochester
Professor, Department of Neurology, University of Rochester
Professor, Department of Pathology and Laboratory Medicine, University of Rochester
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Dr. Tawil's current research interest in focused on FSHD and he is leading an international collaborative effort to understand the underlying molecular mechanisms in FSHD and work towards treatments to slow progression and improve strength in FSHD. Dr. Tawil also participated in a number of research studies that helped identify the genetic cause for the different forms of periodic paralysis. More broadly, Dr. Tawil is interested in bringing new treatments to neuromuscular disorders and has participated and helped organize numerous neuromuscular treatment trials.
Clinic
Dr. Tawil's clinic is located at the University of Rochester Medical Center.
Laboratory website▸
Contact Information
Rabi_Tawil@urmc.rochester.edu | (585) 275-2559
Strong Memorial Hospital
601 Elmwood Ave, AC-1
Rochester, NY 14642
Member
Leo H. Wang, M.D., Ph.D.
Assistant Professor, Department of Neurology, University of Washington
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Dr. Wang's subspeciality training has given him expertise in helping patients with the diagnosis and treatment of neuromuscular diseases including amyotrophic lateral sclerosis (Lou Gehrig's disease), myasthenia gravis, peripheral neuropathies (especially Guillain-Barre syndrome and chronic inflammatory demyelinating polyneuropathy), inherited neuropathies and myopathies, inflammatory myopathies, myotonic disorders, and muscular dystrophies. He also has an interest in autoimmune encephalitis.
Clinic
Dr. Wang's clinic is at the Neurology Clinic at the University of Washington Medical Center.
Laboratory website▸
Contact Information
leowang@uw.edu | (206) 598-7688
1959 NE Pacific Street
Box 356465
Seattle, Washington 98195
Member
Jeffery M. Statland, M.D.
Assistant Professor, Department of Neurology, University of Kansas Medical Center
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Dr. Statland's research background has centered primarily on describing the natural history of and response to therapy for neuromuscular diseases. He completed a neuromuscular fellowship in experimental therapeutics of neurological diseases at the University of Rochester Medical Center, and currently serves as principal investigator or co-investigator for research studies in Facioscapulohumeral Muscular Dystrophy (FSHD), Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, and Myotonic Dystrophy.
Clinic
Dr. Statland's clinic is at the Neurology Clinic at the University of Kansas Medical Center.
Laboratory website
Contact Information
jstatland@kumc.edu
University of Kansas Medical Center
Department of Neurology
Kansas City, KS 66160
Member
Peter Jones, Ph.D.
Associate Professor; Mick Hitchcock Endowed Chair of Medical Biochemistry
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Drs. Peter and Takako Jones function as co-PIs, combining expertise in gene regulation, biochemistry, and chromatin with expertise in cell, developmental, and molecular biology. Together we investigate epigenetic mechanisms of gene regulation, focusing on the combined roles of DNA methylation, histone post-translational modifications, active and repressive chromatin regulators, and noncoding RNAs.
We use a wide range of approaches, including biochemistry, cell biology, developmental biology, proteomics, and epigenomics, to investigate the epigenetic mechanisms that function during normal muscle development and establish and maintain disease states. Our experimental approaches have utilized several different model organisms including mouse, Drosophila, and primary human cell culture models and in vitro systems. Overall, we aim to design therapeutic approaches targeting the epigenetic regulatory mechanisms involved in human disease with a particular focus on myopathies.
Laboratory
Dr.Jones's lab is at the Center for Molecular Medicine at the University of Nevada, Reno School of Medicine.
Laboratory website
Contact Information
peterjones@med.unr.edu l (775)784-1566
1664 North Virginia Street Center for Molecular Medicine Reno, NV 89557