Jeffrey S. Chamberlain, Ph.D.
Professor, Department of Neurology, Medicine and Biochemistry
University of Washington

Dr. Chamberlain is a geneticist with expertise on DMD and gene therapy, is the Center Director, Principal Investigator of
Project 1 and will assist in the design and implementation of preclinical studies as well as the overall
management and training of research personnel involved in preclinical studies. He is also P.I. of the Vector
Core. He will supervise the direction and goals of the Center.

Stephen J. Tapscott, M.D., Ph.D.
Associate Professor, Department of Neurology
University of Washington, Fred Hutchinson Cancer Research Center

Dr. Tapscott, a board certified neurologist specializing in neurogenetics and neuromuscular disease, is the
Center Co-Director and will assist in the creation and implementation of the clinical trials as well as supervise
the trial and the other neurologists involved.

Rainer Storb, M.D.
Professor of Medicine/Program Head, Transplant Biology
University of Washington, Fred Hutchinson Cancer Research Center

Dr. Storb has extensive experience in canine and human stem cell transplantation, and is one of the
leading transplant surgeons in the world. He will provide advice on canine transplant studies, pre-clinical
testing of dystrophin delivery in the cxmd dog, and help guide the mixed chimerism studies and the in vivo
portion of the stem cell transfer studies

James M. Allen, Ph.D.
Senior Research Scientist/Engineer
University of Washington

Dr. Allen is an expert at AAV vectors, including production and use in pre-clinical studies of gene therapy.
Dr. Allen is director of our Vector Core.

Stephen D. Hauschka, Ph.D.
Professor of Biochemistry
University of Washington

Dr. Hauschka is the one the world’s leading muscle cell biologist who has published extensively on
myogenic cell cultures, muscle development and muscle gene expression. He has a particular interest in
developing regulatory cassettes for human gene therapy applications, and is co-investigator on Project 1.

Thomas Bird, M.D.
Professor, Department of Neurology
University of Washington, Veterans Affairs Medical Center

Dr. Bird is a neurogeneticist who has published widely on genetic diseases of the nervous system,
including Alzheimer’s disease, Huntington’s disease, Charcot-Marie-Tooth neuropathy and the hereditary
ataxias. He has a special interest in the provision of high quality genetic counseling for families with
neurogenetic diseases and the delineation of ethical issues in genetic testing. He is the former director of our
MDA clinic, and will be director of the Diagnostic and Genetic Counseling Core.

Sidney M. Gospe, Jr. M.D., Ph.D.
Professor of Neurology and Pediatrics
University of Washington

Dr. Gospe is the Neurology director for the MDA Clinic at Children's Hospital and sees a large number of children
with muscular dystrophy and other genetic myopathies. He is also a co-investigator with the the Diagnostic
and Genetic Counseling Core and will assist with the clinical trial.

Paul Gregorevic, Ph.D.
Acting Assistant Professor, Department of Neurology
University of Washington

Dr. Gregorevic employs a variety of physiological techniques in conjunction with the development and
application of recombinant viral vectors to study the mechanisms of skeletal muscle adaptation, and to develop
prospective approaches for the treatment of muscle diseases. Additionally, Dr. Gregorevic helps to maintain
the mouse physiology core facility at the University of Washington. He will assist with Project 1, Aim 1.

Ross M. Hays, M.D.
Professor, Depts. of Rehabilitation Medicine and Pediatrics
University of Washington, Children's Hospital and Regional Medical Center

Dr. Hays, board certified in pediatrics and physical medicine and rehabilitation, is director of the MDA clinic
at Children’s Hospital and will be assist with evaluation and management of the M.D. patients in the Diagnostic
and Genetic Counseling Core and with the phase 1 clinical gene therapy trial.

Martin J. Kushmerick, M.D., Ph.D.
Professor of Radiology
University of Washington

Dr. Kushmerick is expert in muscle cell mechanics and energy metabolism, and in the use of MRI imaging
and spectroscopy to investigate physiology and pathophysiology of muscle including human muscular
diseases. He holds joint appointments in Bioengineering and in Physiology. He is a co-investigator on Project 2.

Dennis W. W. Shaw, M.D.
Professor, Department of Radiology / Staff Radiologist Children's Hospital and Regional Medical Center
University of Washington & Children's Hospital and Regional Medical Center

Dr. Shaw is an expert in interventional radiology with a particular interests in imaging and spectroscopy of
neurologic and neuromuscular developmental disorders. He will lead the studies on MRI imaging of DMD
patients.

Seth D. Friedman Ph.D.
Research Associate Professor, Department of Radiology
University of Washington

Dr. Friedman has extensive experience developing novel magnetic resonance spectroscopy and imaging
techniques for quantifying changes within diseases that range from autism, bipolar and panic disorder, lupus
and traumatic brain injury.

Wylie Burke, M.D., Ph.D.
Professor and Chair, Department of Medical History and Ethics
University of Washington

Dr. Burke is a world renowned expert in ethical issues related to genetics research. She is Principal
Investigator of the University of Washington Center for Genomics and Healthcare Equality, an NIH-funded
Center of Excellence in Ethical, Legal, and Social Implications (ELSI) Research. Her research addresses the
social, ethical and policy implications of genetic information, and is a key advisor to or Wellstone Center.

Stanley Froehner, Ph.D.
Professor and Chair, Department of Physiologyl and Biophysics
University of Washington

Dr. Froehner is a well known expert in the analysis of the dystrophin-glycoprotein complex and will provide
guidance in testing the gene therapy vectors for Phase 1 clinical trials as well as with the analysis of DGC
expression in dogs in the preclinical and clinical trials.

Marvin E. Adams, Ph.D.
Res Assoc Professor, Department of Physiology and Biophysics
University of Washington

Dr. Adams is a molecular biologist developing and analyzing animal models of muscular dystrophy and
neuromuscular disease.

Joshua O. Benditt, M.D.
Professor and Adjunct, Medicine/Division of Pulmonary and Critical Care/Rehabilitation Medicine
University of Washington

Dr. Benditt is a pulmonologist who sees patients in the MDA clinic and does clinical research related to
respiratory status in the muscular dystrophies including best measurements of function, noninvasive ventilation
and other ventilatory intervention.

Louis J. Boitano, M.S., R.R.T.
Respiratory Care Practitioner, Pulm/Med Spec Clinic
University of Washington

Dr. Boitano's interest in neuromuscular disease is centered on the respiratory support of neuromuscular
patients with respiratory compromise. He focuses on noninvasive respiratory support of MD patients and is
interested in diagnostic tools that can improve the timeliness of starting therapy and providing optimal therapy
support.

Donald Born, M.D., Ph.D.
Associate Professor, Department of Pathology
University of Washington, Harborview Medical Center

Dr. Born is director of the Muscle Biopsy Pathology Lab at the University of Washington and Harborview Medical Centers. He also presents a monthly conference reviewing muscle biopsies for neurologists seeing patients with
neuromuscular diseases at the MDA clinics.

Gregory T. Carter, M.D., MS
Professor, Department of Rehabilitation Medicine
University of Washington

Dr. Carter is a physical medicine and rehabilitation specialist (physiatrist) who co-directs the Muscular
Dystrophy Association/Amyotrophic Lateral Sclerosis (ALS) Clinics at the University of Washington Medical
Center. His clinical research is focused on trying to better understand the relationship between pain, physical
disability, and quality of life in neuromuscular disease (NMD). This would allow for the development of better
treatment protocols, ultimately improving the rehabilitation and palliative care of this patient population. His
pre-clinical research involves characterizing the neuromuscular electrophysiology of genetically corrected
animal models of human NMD.

Kevin E. Conley, Ph.D.
Professor, Depts of Radiology and Physiology & Biophysics
Adjunct Professor of Bioengineering
University of Washington

Dr. Conley's focus is on muscle energetics and metabolism using non-invasive methods (magnetic
resonance & optics) along with computational and analytic tools to quantify the ATP, O2 and H+ fluxes in
active muscle. These measurements allow evaluation of the regulation and integration of energy supply with
demand in normal muscle and the change in muscle energetics with disease.

Joel Chamberlain, Ph.D.
Research Assistant Professor, Department of Medicine
University of Washington

Dr. Chamberlain was our Center’s Wellstone Fellowship recipient and is now a faculty member in Medical
Genetics. Dr. Chamberlain’s lab studies dominantly inherited genetic disorders, and is exploring shRNA and
miRNA delivery in conjunction with myotonic dystrophy and FSHD.

Phil Chance, M.D., Ph.D.
Professor Depts. of Pediatrics and Neurology
University of Washington, Children's Hospital Regional Medical Center

Dr. Chance is a medical geneticist with great interest in peripheral neuropathies. He has cloned several
genes responsible for Charcot-Marie-Tooth Disease and is Chief of the Division of Genetics and Development
at Children’s Hospital.

David Dichek, M.D.
John L. Locke Professor of Medicine
University of Washington

Dr. Dichek is chief of the Division of Cardiology at the University of Washington Medical Center. He is an expert in the field of cardiovascular gene therapy. He has a number of collaborations with the labs of Jeff Chamberlain and Chuck
Murry regarding gene therapy strategies. Most of his group’s work is targeted at cardiac blood vessels, but he
is also interested in gene transfer to the heart.

Galina Filippova, Ph.D.
Assistant Member
Fred Hutchinson Cancer Research Center

Dr. Filippova studies chromosomal boundaries and the mechanism that control the separations between
inactive and active sets of genes on chromosomes. She discovered that the protein CTCF can form
boundaries that protect entire chromosomal domains. She has a Wellstone Pilot project on the analysis of
chromatin structure and CTCF in FSHD.

Brian K. Kennedy, Ph.D.
Assoc Professor of Biochemistry
University of Washington

Professor Kennedy is an expert in nuclear lamins and the genetics and biochemistry of cellular aging. He
and Dr. Hauschka collaborate closely on studies of Emery-Dreifuss Muscular Dystrophy and
Fascioscapulohumeral Muscular Dystrophy. Their EDMD studies focus on determining how nuclear lamin and
emerin mutations and deficiencies affect skeletal and cardiac muscle gene transcription. Their FSHD studies
focus on identifying the molecular functions of FRG-1, a protein whose possible over-expression in FSHD may
be partially responsible for FSHD pathology.

Michel Kliot, M.D.
Professor, Department of Neurosurgery
University of Washington

Dr. Kliot is a neurosurgeon with special expertise in the neuromuscular area. He has a special interest in
MRI evaluation of nerve and muscle disorders.

George H. Kraft, M.D., M.S.
Professor, Department of Rehabilitation Medicine
University of Washington

Dr. Kraft is board certified in Physical Medicine and Rehabilitation, and co-director of the MDA clinic at the
University of Washington Medical Center.

Albert R. La Spada, M.D., Ph.D.
Associate Professor, Depts. of Lab Medicine, Medicine, Neurology & Pathology
Director, Center for Neurogenetics & Neurotherapeutics
University of Washington

Dr. La Spada is an internationally renowned neurodegenerative disease researcher whose interests include
polyglutamine repeat disorders. His lab is focused on the molecular basis of the motor neuron degeneration in
the neuromuscular disease, X-linked spinal and bulbar muscular atrophy (SBMA). Dr. La Spada's group is
collaborating with Dr. Chamberlain and other members of the Center in an effort to develop methods for gene
delivery of growth factors and other genes to motor neurons in a mouse model of SBMA.

Mark Lewin, M.D.
Assistant Professor, Department of Pediatrics
Children's Hospital and Regional Medical Center

Dr Lewin's interests center around patient care, i.e., understanding the efficacy of currently accepted
therapeutic strategies for treating the dilated cardiomyopathy associated with MD. To this end, he sees the
majority of patients with MD referred for either cardiac evaluation or management at Children's Hospital. He is
very interested in exploring the influence of novel clinical therapies, and the cardiac evaluation of animal models via echocardiography to assess left ventricular functional parameters.

Andre Lieber, Ph.D.
Professor, Department of Medicine
University of Washington

Dr. Lieber is an expert in the area of gene therapy using adenoviral vectors. He has published extensively on
vector toxicology and immune responses. He has a Wellstone pilot project studying AAV6 trophism for blood
cells and innate immune response profiles.

A. Dusty Miller, Ph.D.
Member, Fred Hutchinson Cancer Research Center

Dr. Miller is one of the world’s leading experts in the field of gene therapy, having pioneered development
of both retroviral and AAV vector technology. He is a former member of the NIH Recombinant DNA Advisory
Committee (RAC), and the American Society of Gene Therapy’s Board of Directors. Locally, Dr. Miller directs
the Molecular Therapy Core Center, and will closely collaborate with the Chamberlain Lab and the Vector Core
in developing AAV6 vector technology. He will also be an advisor to our Center’s Executive Committee.

Daniel G. Miller, Ph.D., M.D.
Asst Professor, Pediatrics
Adjunct Member, Department of Genome Sciences
University of Washington

Dr. Miller is a Pediatric Geneticist at Children’s Hospital. Dr. Miller’s lab is testing the hypothesis that Dux4
gene expression is altered in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) in an attempt to
understand how muscle strength is compromised by chromosome 4 repeat-array contractions that cause this
condition. Dr. Miller is also investigating gene therapy strategies that utilize targeted gene-repair as a
mechanism for disease reversal.

Charles E. Murry, M.D., Ph.D.
Associate Professor, Department of Pathology
University of Washington

Dr. Murry's laboratory is interested in diseases of the myocardium, particularly myocardial infarction and
infarct repair. They study both normal infarct repair mechanisms (angiogenesis, granulation tissue formation,
scar formation) and possible cell and gene therapies to enhance infarct repair and have used committed cells
such as skeletal and cardiac myocytes and more recently have made a major commitment to the study of adult
and embryonic stem cells for infarct repair. Many of the strategies they are exploring for infarct repair would be
applicable to patients with muscular dystrophy-related cardiomyopathy, and some likely would be applicable to
direct regeneration of skeletal muscle in dystrophic patients.

Robert Pearlman, M.D., M.P.H.
Professor, Department of Medicine
University of Washington

Dr. Pearlman is a Medical Ethicist who is interested in the field of Gene Therapy. He is in the division of
Division of Gerontology and Geriatrics and will serve as an advisor to our Executive Committee.

David Russell, M.D., Ph.D.
Associate Professor, Department of Medicine
University of Washington

Dr. Russell is an expert on gene targeting and the use of AAV vectors. He discovered AAV6 and
pioneered its use in collaboration with the Miller Lab and with Avigen. He will be an advisor to our Center’s
Executive Committee and is collaborating with the Chamberlain Lab on developing AAV6 technology.

Ron Scott, M.D.
Professor, Department of Pediatrics
University of Washington

Dr. Scott is a member of the division of Genetics and Development and has a particular interest in DNA
diagnostics. Dr. Scott heads the diagnostics unit for Children’s Hospital and directs the molecular diagnosis of
our muscular dystrophy patients.

Barbara Trask, Ph.D
Member, Fred Hutchinson Cancer Research Center
Professor of Genome Sciences, University of Washington

The Trask group studies large-scale facets of genome organization. Her work relies on continued
development of fluorescence in situ hybridization (FISH), a means of fluorescently tagging specific DNA
sequences in chromosomes or nuclei, and flow cytometry, a technology for isolating specific chromosomes for
molecular analyses based on their DNA content. Dr. Trask has a Wellstone pilot project studying nuclear
organization in FSHD.

Michael D. Weiss, M.D.
Associate Professor, Department of Neurology
University of Washington

Dr. Weiss is a neuromuscular neurologist with a strong interest in myopathies of late onset, in particular
myofibrillar myopathy and inclusion body myositis. He is currently involved in research investigating the role of
presenilin 1 and 2 in the pathogenesis of inclusion body myositis as well as research utilizing various
electrophysiologic techniques to characterize mouse models of neuromuscular disease.

Sara J. Wiyrick, M.S., C.G.C.
University of Washington

Sara Wiyrick works as a genetic counselor at the University of Washington Medical Center. Her primary
responsibilities include providing risk assessment and genetic counseling for individuals and families seen in
the Neurogenetics Clinic, Muscular Dystrophy Association Clinic and Medical Genetics Clinic. Sara earned her
Master of Science degree in Human Genetics from the University of Michigan Genetic Counseling Training
Program in 2001 and was certified by the American Board of Genetic Counseling in 2002.

Zipora Yablonka-Reuveni, Ph.D.
Professor, Department of Biological Structure
University of Washington

Dr. Yablonka-Reuveni is a renowned leader in the field of skeletal muscle stem cells. Her research goal is
to gain insights into the regulation of satellite cell recruitment and renewal during growth, aging and disease.
She also investigates the contribution of vasculature-derived cells to myogenesis and the origin of
mesenchymal stem cells, residing in the muscle tissue.