Donald R. Wassenberg II

Diana Bautista, Ph.D.
Professor and Head of the Division of Cell Biology, Developmental Biology and Physiology
Department of Molecular and Cell Biology and the Helen Wills Neuroscience Institute
University of California, Berkeley
http://www.cincinnatichildrens.org/research/divisions/m/mcb/labs/robbins/default/

Peripheral Neuroimmune Signaling in Eczema: Deciphering Roles of Human Disease Genes in Skin and Airway Inflammation
Dr. Bautista is a Howard Hughes Medical Institute Investigator, Professor, and Head of the Division of Cell Biology, Developmental Biology, and Physiology in the Department of Molecular and Cell Biology and the Helen Wills Neuroscience Institute at the University of California, Berkeley. She received her bachelor’s degree in Biology & Biochemistry from the University of Oregon, working with Dr. Peter O’Day studying visual transduction in the fly. She pursed her Ph.D. in Neuroscience at Stanford University, working with Dr. Richard S. Lewis on understanding calcium dynamics in human T lymphocytes. Her postdoctoral research with Dr. David Julius in the Department of Physiology at the University of California, San Francisco focused on defining the physiological roles of sensory ion channels TRPA1 and TRPM8 in thermosensation and pain. She joined the faculty at UC Berkeley in 2008. Dr. Bautista’s lab studies the neuroimmune interactions underlying chronic pain, itch and airway inflammation. Her research has been funded by the NIH since 2009 and she is a recipient of a Pew Scholar Award, the Society for Neuroscience Young Investigator Award, an NIH Director’s Transformative Research Award, and UC awards for Graduate Mentoring for her work in Diversity, Equity and Inclusion in STEM.

Nancy Bonini, Ph.D.
Florence R.C. Murray Professor
Department of Biology, University of Pennsylvania
https://www.bio.upenn.edu/people/nancy-bonini

Watch Dr. Bonini’s Talk

Drosophila as a Model for Human Neurodegenerative Disease
Dr. Bonini is the Florence R.C. Murray Professor of Biology in the Department of Biology (School of Arts and Sciences) at the University of Pennsylvania and faculty in the Cell & Developmental Biology Department in the Perelman School of Medicine, with affiliations with the Cell and Molecular Biology (CAMB) and Neuroscience Graduate Groups (NGG).

Beginning in her post-doctoral studies under Dr. Seymour Benzer, Dr. Bonini focused on using the fly as a tool for understanding the genetic basis of the brain and behavior. Since establishing her lab at Penn in 1994, she has continued to make significant contributions in the fields of genetics, molecular biology and degeneration using the fly to elucidate mechanisms of human neurodegenerative disease, brain injury and aging.

Her pioneering work in this field has resulted in numerous accolades including a David and Lucile Packard Award, John Merck Scholars Award, being named an investigator of the Howard Hughes Medical Institute and an NIH R35 Outstanding Investigator award.

Dr. Bonini is also an elected member of the National Academy of Science, an elected member of the National Academy of Medicine, an elected member of the American Academy of Arts and Sciences, and an elected fellow of the American Association for Advancement of Science.

Paul Turner, Ph.D.
Professor of Microbiology
Department of Biology, University of Pennsylvania
https://medicine.yale.edu/profile/paul_turner

Watch Dr. Turner’s Talk

Phage therapy to combat antibiotic-resistant bacterial infections in cystic fibrosis patients
Dr. Paul Turner is the Rachel Carson Professor of Ecology and Evolutionary Biology at Yale University and a Microbiology faculty member at Yale School of Medicine. He obtained a BA in Biology (1988) from the University of Rochester, a Ph.D. in Microbial Evolution (1995) from Michigan State University, and did postdoctoral work at the National Institutes of Health, the University of Valencia in Spain, and the University of Maryland-College Park, before joining Yale in 2001.

Dr. Turner studies the evolutionary genetics of viruses, particularly phages (bacteria-specific viruses) that infect bacterial pathogens and RNA viruses transmitted by mosquitoes. He researches the use of phages to treat antibiotic-resistant bacterial diseases.

Dr. Turner is very active in science-communication outreach to the general public, and is involved in programs where faculty members collaborate with K-12 teachers to improve STEMM education in underserved public schools.

Dr. Turner’s service includes the National Science Foundation’s Bio Advisory Committee and he is President-elect of the International Society for Evolution, Medicine and Public Health. His honors include Fellowship in the National Academy of Sciences, the American Academy of Arts & Sciences, and the American Academy of Microbiology.

Madelon Maurice, Ph.D.
Professor of Molecular Cell Biology
Center for Molecular Medicine, University Medical Center Utrecht, The Netherlands
https://www.uu.nl/en/research/life-sciences/madelon-maurice

Watch Dr. Maurice’s Talk

Mechanisms of Wnt pathway tumor suppressor mutations in cancer: beyond loss-of-function
Madelon Maurice is Professor of Molecular Cell Biology at the University Medical Center Utrecht, The Netherlands, from 2016 and a member of the national Oncode Institute for cancer research. She earned her Ph.D. (1998) at Leiden University, The Netherlands and performed postdoctoral work in the laboratories of Hidde Ploegh, Harvard Medical School, USA, and Hans Clevers, Hubrecht Institute, The Netherlands.

Her primary research interests are to uncover the mechanisms by which cells communicate during tissue renewal and how these processes are subjected to faulty regulation during human cancer development.

Her multidisciplinary research team is amongst the leading laboratories internationally in the field of Wnt signaling, a major signaling pathway that controls the maintenance and activity of stem cell reservoirs and is frequently misused by cancer cells.

The innovative nature of her work led to the award of an ERC Starting grant (2009), a VICI grant from the Netherlands Organization for Scientific Research (NWO; 2015) and election as a member of the national Oncode Institute (2017). She is co-chair of the Utrecht Platform for Organoid Technology.

Leslie Leinwand, Ph.D.
Distinguished Professor of Molecular, Cellular and Developmental Biology
Center for Molecular Medicine, University Medical Center Utrecht, The Netherlands
Chief Scientific Officer
BioFrontiers Institute at the University of Colorado Boulder
https://www.colorado.edu/mcdb/leslie-leinwand

Diseases Caused by Myosin Mutations: Pathogenesis and Potential Therapies
Leslie Leinwand, Ph.D., is a Molecular, Cellular, and Developmental Biology (MCDB) Distinguished Professor and the Chief Scientific Officer of the BioFrontiers Institute at the University of Colorado Boulder. She was recruited to be Chair of MCDB in 1995. She received her bachelor’s degree from Cornell University and her Ph.D. from Yale University, and she did post-doctoral training at Rockefeller University. She joined the faculty at Albert Einstein College of Medicine in New York in 1981 and remained there until moving to Colorado in 1995. She co-founded Myogen, Inc., which was sold to Gilead Pharmaceuticals. More recently, she co-founded Hiberna, Inc., and MyoKardia, Inc., a company founded to develop therapeutics for inherited cardiomyopathies. She is a Fellow of the AAAS, former MERIT Awardee of the NIH, Established Investigator of the American Heart Association, and was recently elected to the American Academy of Arts and Sciences and the National Academy for Inventors. She has been honored by the American Heart Association with its Distinguished Scientist Award. The interests of Dr. Leinwand’s laboratory are the genetics and molecular physiology of inherited diseases of the heart and how gender and diet modify the heart. The study of these diseases has required multidisciplinary approaches involving molecular biology, mouse genetics, mouse cardiac physiology, and the analysis of human tissues.

Paul Negulescu, Ph.D.
Senior Vice President, Research and San Diego Site Head
Vertex Pharmaceuticals Incorporated
https://www.vrtx.com/leadership/paul-negulescu-phd

Can We Fix the Broken Protein that Causes Cystic Fibrosis?
Dr. Paul Negulescu received both his B.S. and Ph.D. from U.C. Berkeley in physiology and carried out post-doctoral work at U.C. Berkeley and U.C. Irvine in the areas of biophysics and immunology. He is responsible for leading the Vertex San Diego research site, which employs approximately 220 people. Under his leadership, the San Diego site has produced multiple novel drug candidates which have become approved medicines. One of these, Kalydeco, was approved in January 2012 as the first drug to treat the underlying cause of cystic fibrosis (CF). A second drug, Orkambi, was approved in July 2015 to treat the most common form of CF. Both Kalydeco and Orkambi are recognized as successful examples of personalized medicine. Vertex’s CF program was conducted in collaboration with the Cystic Fibrosis Foundation, and is regarded as a model for biotech collaboration with disease-focused advocacy groups.

Joseph C. Wu, M.D., Ph.D.
Director of Stanford Cardiovascular Institute
Professor of Medicine and Radiology
https://profiles.stanford.edu/joseph-wu

Cardiac Induced Pluripotent Stem Cells for Precision Medicine
Dr. Joseph Wu is Director of the Stanford Cardiovascular Institute and Professor in the Department of Medicine (Cardiology) and Department of Radiology (Molecular Imaging Program). He received his M.D. from Yale and completed his medicine residency and cardiology fellowship training followed by a Ph.D. in molecular pharmacology at UCLA. His awards include the Burroughs Wellcome Foundation Career Award in Medical Sciences, Baxter Foundation Faculty Scholar Award, NIH Director’s New Innovator Award, NIH Roadmap Transformative Award and the Presidential Early Career Award for Scientists and Engineers presented by President Obama. Dr. Wu is on the editorial board of the Journal of Clinical Investigation, Circulation Research and several other journals. Dr. Wu’s clinical activities involve adult congenital heart disease and echocardiography. His lab studies the biological mechanisms of adult stem cells, embryonic stem cells, and induced pluripotent stem cells. Approaches to better understand stem cell biology include next generation sequencing, tissue engineering, physiological testing and molecular imaging technologies. His group uses induced pluripotent stem cells for cardiovascular disease modeling, drug screening and cell therapy. They also develop vectors and therapeutic genes for cardiovascular gene therapy applications.

Forest Rohwer, Ph.D.
Department of Biology at San Diego State University
http://coralandphage.org/index.php

Developing a Personalized Medicine Platform for Cystic Fibrosis
Forest Rohwer is a Fellow of the American Academy for Advancement of Science (AAAS), American Academy of Microbiology (AAM) and Canadian Institute for Advanced Research (CIFAR). He led the development of viromics, which involves isolating and sequencing the RNA/DNA from all of the viruses in a sample. From this data, it is possible determine what types of viruses are present and what functions they are encoding. Dr. Rohwer uses viromics to study ecosystems ranging from the human body to coral reefs and has shown that most genomic diversity on the planet is viral. By applying these approaches to the cystic fibrosis lung, his lab has shown that much of the microbiology in this disease has been missed. These insights are being translated into personalized knowledge about individual patient’s disease state and potential treatments. Dr. Rohwer has published >150 peer-reviewed articles, was awarded the International Society of Microbial Ecology Young Investigators Award in 2008, and was listed as one of the World’s Most Influential Scientific Minds in 2014. He has also published two books: Coral Reefs in the Microbial Seas and Life in Our Phage World.

John M. Carethers, M.D.
John G. Searle Professor and Chair
Department of Internal Medicine at the University of Michigan
http://www.uofmhealth.org/profile/1238/john-m-carethers-md

The multiple facets of DNA mismatch repair defects and their influence on hereditary colorectal cancer.
Dr. Carethers received both the B.S. and M.D. at Wayne State University. He completed his internship and residency in internal medicine at Massachusetts General Hospital. Subsequently he performed a fellowship in gastroenterology and postdoctoral research at the University of Michigan. Dr. Carethers served on the faculty in medicine at UCSD from 1995-2009. His research focuses on colorectal cancer, with interests in mechanisms of tumor progression, tumor genetics and markers, familial cancer and polyposis syndromes, DNA mismatch repair, molecular pathology, TGFβ superfamily signaling in cancer progression and colorectal cancer disparities. Dr. Carethers continues to have significant funding from the National Institutes of Health and has received numerous honors, including election to the Institute of Medicine of the National Academy of Sciences. He is Senior Associate Editor for the journal Gastroenterology and serves on several scientific advisory boards. Dr. Carethers has authored over 100 peer-reviewed scientific publications.

Mario R. Capecchi, Ph.D.
Department of Human Genetics, University of Utah School of Medicine
http://capecchi.genetics.utah.edu/mario/

Gene Targeting into the 21st Century: Mouse Models of Human Disease from Cancer to Neuropsychiatric Disorders.
Dr. Capecchi is best known for his pioneering work on the development of gene targeting in mouse embryo-derived stem (ES) cells. This technology allows scientists to create mice with mutations in any desired gene. The power of this technology is that the investigator chooses both which gene to mutate and how to mutate it. The investigator has virtually complete freedom on how to manipulate the DNA sequences in the genome of living mice. This allows scientists to evaluate in detail the function of any gene during the development or post-developmental phase of the mouse. His research interests include the molecular genetic analysis of early mouse development, neural development in mammals, production of murine models of human genetic diseases, gene therapy, homologous recombination and programmed genomic rearrangements in the mouse.

Nigel G. Laing, Ph.D.
Head of Neuromuscular Diseases at the Centre for Medical Research University of Western Australia
https://www.perkins.org.au/our-people/laboratory-heads/laing-profile/

Next generation DNA sequencing: Gene discovery, improved diagnostics and disease prevention.
Nigel G. Laing Ph.D. is head of Neuromuscular Diseases at the Centre for Medical Research University of Western Australia. He received his Ph.D. from Edinburgh University in 1979. In his Ph.D. and early postdoctoral studies (up to 1987) he was a neuroembryologist working on factors that control motor neuron death and muscle fibre type. In 1987 – 1988 he retrained in human molecular genetics with Allen Roses at Duke University. Returning to Western Australia, he developed research and diagnostic molecular neurogenetics laboratories. His laboratory has identified 20 human disease genes including slow alpha-tropomyosin as the first gene for nemaline myopathy; skeletal muscle alpha actin as a significant cause of congenital myopathies, slow skeletal/beta cardiac myosin as the cause of “Laing” distal myopathy and recently mutations in KLHL40 as a major cause of myopathic fetal akinesia.

K. David Becker, Ph.D.
Senior Director, Lab Operations, Helix, San Diego, CA
http://www.helix.com/our-team/

Translational Science, Clinical Medicine and Personal Genomics
K. David Becker Ph.D., MB (ASCP) is chief scientific officer of Pathway Genomics Corporation, where he is founding scientist and responsible for clinical lab and FDA regulatory affairs. Dr. Becker received a B.A. from the University of California, Santa Barbara and a Ph.D. from the SDSU/UCSD Joint Doctoral Program (in 1991). He performed analysis of human cardiomyopathy models for his postdoctoral work at UCSD with support from American Heart Association and National Institutes of Health fellowships. He subsequently served as director of a mouse genetics core facility at UCSD. Dr. Becker then established himself as a research scientist in the biotechnology industry, studying gene expression and genomics. He began at MitoKor and moved to Neurogenetics, which became TorreyPines Therapeutics. He helped found Pathway Genomics in 2008. Dr. Becker oversees the company’s scientific development as well as compliance issues. In addition to publications on his early muscle-based work, Dr. Becker has published numerous papers on genomics, with a particular focus on the genetic basis of Alzheimer’s disease.

Albert La Spada, M.D., Ph.D.
Head, Division of Genetics, Department of Pediatrics, UC San Diego School of Medicine, La Jolla, CA
http://laspadalab.ucsd.edu/Pages/default.aspx

Dynamic Mutation and Human Disease: Insights into Neurodegenerative Repeat Expansion Disorders
Albert La Spada, M.D., Ph.D., FACMG is professor of Pediatrics and of Cellular & Molecular Medicine at the University of California, San Diego since 2009. Dr. La Spada serves as Division Head of Genetics and is a founding member of the Institute for Genomic Medicine. He received M.D. and Ph.D. training at the University of Pennsylvania School of Medicine. His dissertation on Kennedy’s disease pinpointed the expansion of a trinucleotide repeat in the androgen receptor gene as the cause of this neurodegenerative disorder. Dr. La Spada pursued clinical and postdoctoral training at the University of Washington Medical Center and joined the faculty there in 1998. Dr. La Spada’s research laboratory focuses on the molecular basis of neurodegenerative disease. The group is probing the molecular basis of neurodegeneration and neuron cell death in spinocerebellar ataxia type 7, and has found connections between pathways involved in transcription and neuron dysfunction. By reproducing molecular pathology in model organisms, they have also begun to develop therapies. Dr. La Spada has been funded by the NIH, Howard Hughes Medical Institute, Muscular Dystrophy Association and other agencies. He has received several scientific awards and sits on a variety of editorial boards and grant review committees.

Atul Butte, M.D., Ph.D.
Professor, UCSF School of Medicine
http://profiles.ucsf.edu/atul.butte

Exploring Genomic Medicine Using Translational Bioinformatics
Atul Butte, M.D., Ph.D. is assistant professor of Medical Informatics and Pediatrics at Stanford University School of Medicine and holds a concurrent appointment in Computer Science. Dr. Butte received an A.B., M.S. and M.D. from Brown University, as well as a M.S. in Medical Informatics and a Ph.D. from MIT. Dr. Butte’s laboratory focuses on genomic medicine using translational bioinformatics. These efforts have been applied to cancer drug discovery, type 2 diabetes mellitus, fat cell formation, obesity and transplantation. His lab has developed approaches to automatically index genomic data sets, to re-map microarray data and to compare clinical data from electronic health record systems with gene expression data. Dr. Butte has published in journals from the Nature and Cell groups, Proceedings of the National Academy of Sciences and numerous bioinformatics journals. He sits on several editorial boards and is the scientific founder and a scientific advisor of Genstruct, Inc. (Cambridge, MA), a company focusing on optimizing the pharmaceutical development process and using genomics data to enable individualized medicine.

Garry P. Nolan, Ph.D.
Professor of Microbiology and Immunology, Stanford University, Stanford, CA
http://www.stanford.edu/group/nolan/

Phospho-Biosignatures and Disease
Dr. Garry P. Nolan is Associate Professor of Microbiology and Immunology at Stanford University School of Medicine. His laboratory group has recently focused on the analysis of biological events at the single cell level using novel genetic and FACS-based approaches, particularly application of proteomics to understanding immune signaling. They have also engineered viral cloning and expression systems that are in use worldwide. Dr. Nolan received a Ph.D. in 1989 from Stanford University and performed postdoctoral work at MIT with Dr. David Baltimore. He has published over 125 journal articles and serves on the editorial boards of three journals. Dr. Nolan is the recipient of the Stohlman Scholar Award of the Leukemia and Lymphoma Society (the Society’s highest award) and a Burroughs Wellcome Award in Pharmacology. He has founded three biotechnology companies, advises several others, and was recently honored as one of the top 25 inventors at Stanford University.

Jeffrey Robbins, Ph.D.
Executive Co-Director of The Heart Institute, Cincinnati Children’s Hospital, Cincinnati, OH
http://www.cincinnatichildrens.org/research/divisions/m/mcb/labs/robbins/default/

Progenitor Cell Populations: the Birth of a Cardiac Syndrome
Dr. Jeffrey Robbins is Professor of Pediatrics and Chair of Molecular Cardiovascular Biology at Cincinnati Children’s Hospital Medical Center. His lab focuses on mammalian cardiac disease at the genetic, cell biological and protein levels. Dr. Robbins received a Ph.D. in 1976 from the University of Connecticut. Prior to moving to Cincinnati in 1985, he served as assistant professor through associate professor at the University of Missouri-Columbia. Dr. Robbins has been an established investigator of the American Heart Association and is now an elected fellow. He received the 2007 Presidential Award for Research from the International Society for Heart Research. He has published over 160 papers in such journals as Nature, Science and Nature Medicine and has served on the editorial boards of 15 journals including the Journal of Biological Chemistry and Circulation Research.

Jeffrey S. Chamberlain, Ph.D.
Director of the Wellstone Muscular Dystrophy Cooperative Research Center, University of Washington, Seattle, WA
http://depts.washington.edu/chamblab/index.html

Development of Gene Therapy for the Muscular Dystrophies
Dr. Jeffrey S. Chamberlain is Professor and Director of the Wellstone Muscular Dystrophy Cooperative Research Center at the University of Washington School of Medicine. His lab analyzes defects induced by genetic forms of muscular dystrophy and develops gene and cell based therapies. Dr. Chamberlain received a Ph.D. in 1985 from the University of Washington and performed postdoctoral research at Baylor College of Medicine. Prior to moving to the University of Washington, he served as assistant professor through professor at the University of Michigan Medical School from 1990-2000. Dr. Chamberlain is a recipient of a MERIT award from the National Institutes of Health and serves on NIH and Muscular Dystrophy Association review panel and advisory boards. He has published over 160 papers in such journals as Nature, Science and Nature Medicine and is on the editorial boards of a number of journals that focus on gene therapy. His collaborations with clinical researchers offer the exciting promise of translating basic science into human therapy.

Andrew D. McCulloch, Ph.D.
Distinguished Professor of Bioengineering, UCSD, La Jolla, CA
http://cmrg.ucsd.edu/AndrewMcCulloch

Cardiac Systems Biology
Dr. Andrew McCulloch is Professor and Chair of Bioengineering at UCSD where he studies experimental and computational cardiac bioengineering and physiology. He received a Ph.D. in 1986 from the University of Auckland, New Zealand in Engineering Science and Physiology. Dr. McCulloch was an NSF Presidential Young Investigator and is a Fellow of the American Institute for Medical and Biological Engineering. He serves on the Board of Directors of the Bio-Medical Engineering Society, as Associate Editor of the Journal of Biomechanical Engineering and on the editorial boards of several other journals. Dr. McCulloch’ group uses experimental and computa­tional models to investigate the relationships between the cellular and extracellular structure of cardiac muscle and the electrical and mechanical function of the whole heart during ventricular remodeling, and arrhythmia. Dr. McCulloch co-founded Insilicomed in 2000.

Christine E. Seidman, M.D.
Professor at Harvard Medical School and Director of the Cardiovascular Genetics Center, Brigham and Women’s Hospital, Boston, MA
http://researchfaculty.brighamandwomens.org/BRIProfile.aspx?id=2340

Genetic Causes of Cardiac Hypertrophy: Rare Disorders or Common Occurrences?
Dr. Christine E. Seidman received her undergraduate degree from Harvard College and earned her medical degree at George Washington University School of Medicine. She was an intern and resident in internal medicine at the Johns Hopkins Hospital. Dr. Seidman received subspecialty training in cardiology at Massachusetts General Hospital. Dr. Seidman is Professor of Genetics and Medicine at Harvard Medical School and Director of the Cardiovascular Genetics Service at Brigham and Women’s Hospital, Boston. She is a leader in research on the genetic basis of heart disease and has dedicated her most recent work to identifying the genetic basis for human cardiomyopathies. Dr. Seidman serves on a number of editorial boards and has published nearly 200 research articles. Dr. Seidman is a recipient of the 2002 Bristol-Myers Squibb Award, which she shared with Jonathan Seidman.

Stuart H. Orkin, M.D.
Chair, Department of Pediatric Oncology, Dana Farber Cancer Institute and Professor of Pediatrics, Harvard Medical School, Boston, MA
http://www.hms.harvard.edu/dms/bbs/fac/orkin.php

Genetic Control of Blood Cell Development
Dr. Stuart H. Orkin has made numerous important contributions to our understanding of gene structure and expression during hematopoietic development. Dr. Orkin received his undergraduate degree from the Massachusetts Institute of Technology. He earned his medical degree at Harvard Medical School, where he continued his clinical training. He pursued research fellowships at the National Institutes of Health and at Harvard Medical School. Dr. Orkin has received numerous professional honors and is a member of the National Academy of Sciences and the Institute of Medicine. He currently serves on the editorial board of eight scientific journals, including Genes and Development and the Journal of Clinical Investigation. Dr. Orkin’s major research interests are hematopoietic development, stem cell biology and leukemia. He has published nearly 300 primary research articles and almost 100 review articles. He is an investigator of the Howard Hughes Medical Institute and David G. Nathan Professor of Pediatrics at Harvard Medical School. Dr. Orkin serves as Chairman of the Department of Pediatric Oncology at Dana Farber Cancer Institute.

Jean Y. J. Wang, Ph.D.
Professor of Medicine, Associate Director of Department of Medicine, Chair of Basic Research, Moores UCSD Cancer Center, La Jolla, CA
http://biology.ucsd.edu/faculty/wang.html

Exploiting an Intrinsic Vulnerability of Uncontrolled Proliferation in Cancer
Jean Y. J. Wang received a B.S. in Botany from the National Taiwan University and a Ph.D. in Biochemistry from UC-Berkeley where she discovered the first protein kinase-dependent regulatory pathway in prokaryotes with Daniel E. Koshland, Jr. She was a Jane Coffin Childs Postdoctoral Research Fellow at the Massachusetts Institute of Technology where she cloned and characterized one of the first human proto-oncogenes in the laboratory of Nobel Laureate David Baltimore. Dr. Wang joined UCSD in 1983 and is associate director of basic research for the UCSD Cancer Center. She also served on the Board of Scientific Counselors at the National Cancer Institute and the Board of Directors of the American Association for Cancer Research. She has received numerous honors and awards, including the Searle Scholar Award in 1984 and the Earl Warren College Outstanding Teacher Award in 1994. She is the Herbert Stern Endowed Chair in Biology. Dr. Wang’s research focuses on elucidating the signaling network that regulates the cellular decision to grow, to differentiate or to commit suicide under genotoxic stress. Her work has identified new links among cancer genes in this network as well as novel biological output from these gene interactions. These findings are directly relevant to the cause of cancer and to the identification of new targets for therapeutic intervention.

Phyllis I. Gardner, M.D.
Professor of Medicine and Clinical Pharmacology, Stanford School of Medicine, Stanford, CA
http://med.stanford.edu/profiles/Phyllis_Gardner/

The Evolving Impact of the Genomic Revolution on Molecular Diagnostics and Therapies
Dr. Phyllis Gardner received a B.S. from the University of Illinois, Urbana in 1972 and a M.D. from Harvard Medical School in 1976. She pursued further clinical and research training at Massachusetts General Hospital, Stanford University Medical Center, Columbia University, and University College in London. Dr. Gardner joined the faculty at Stanford in 1984 and served as Senior Associate Dean for Education and Student Affairs from 1998-2001. Dr. Gardner is a member of the boards of directors and scientific advisory boards of a number of biotechnology firms and was founder of Genomics Collaborative, CambriaTech, and Xeragen. Dr. Gardner’s research interests are in signal transduction, focusing on the role that ion channels play in this process. Her studies include analysis of the channels affected in patients with cystic fibrosis. Dr. Gardner’s numerous research publications include articles in Cell, the Lancet, Science and Nature.

Theodore Friedmann, M.D.
Professor of Pediatrics and Muriel Jeannette Whitehill Chair of Biomedical Ethics, UC San Diego School of Medicine, La Jolla, CA
https://healthsciences.ucsd.edu/som/pediatrics/research/labs/friedmann-lab/pages/default.aspx

Progress Toward Human Gene Therapy: Technology, Policy and Ethics
Dr. Theodore Friedmann received his M.D. from the University of Pennsylvania School of Medicine in 1960. He held positions at Children’s Hospital, Boston, University of Cambridge (UK), Harvard University, and the Salk Institute. He is currently Professor of Pediatrics at the University of California San Diego School of Medicine. In 1993 Dr. Friedmann was appointed Director of the UCSD Program in Human Gene Therapy. He also serves as Muriel Jeannette Whitehill Chair for Biomedical Ethics. Dr. Friedmann has been a member of the Biomedical Ethics Advisory Committee for Congress and the Advisory Committee on Gene Therapy to NIAID/NIH. Dr. Friedmann’s research laboratory focuses on developing efficient methods for transfer of therapeutic genes into defective human cells. The goal is to correct genetic defects underlying human diseases, including cancer. His lab is also developing more efficient methods of producing large amounts of therapeutic gene transfer agents.

Richard Kolodner, Ph.D.
Professor of Medicine, UCSD. Head of the Laboratory of Cancer Genetics, Ludwig Institute for Cancer Research, La Jolla, CA
http://www.ludwigcancerresearch.org/location/san-diego-branch/richard-kolodner-lab

Mutator Genes, Genome Instability and Cancer Susceptibility
Dr. Richard Kolodner is Head of the Laboratory of Cancer Genetics at the San Diego branch of the Ludwig Institute for Cancer Research. He is a Professor of Medicine and Cancer Center Member at the University of California, San Diego. Dr. Kolodner received both a B.S. and Ph.D. in Biological Sciences from UC Irvine. He was a postdoctoral fellow in biological chemistry at Harvard Medical School with Dr. C. C. Richardson from 1975-78. He then joined the Harvard and Dana-Farber Cancer Institute faculty where he rose to professor and head of the division of human genetics. Dr. Kolodner moved to San Diego in 1997. He served on numerous NIH review panels and is an associate editor of Cell. Dr. Kolodner’s research focuses on DNA repair, recombination and replication in yeast and in mammals, with a particular emphasis on understanding how defects in these processes influence cancer susceptibility.

Neal D. Epstein, M.D., Ph.D.
National Heart, Lung, and Blood Institute (Center for Regenerative Medicine), Bethesda, MD

Of Flies and Men: Lessons from the Molecular Biology and Pathophysiology of Hypertrophic Cardiomyopathy
Dr. Neal D. Epstein is Chief of the Molecular Physiology Section, Cardiology Branch, National Heart, Lung and Blood Institute at the National Institutes of Health. He received a B.A. in Philosophy from Clark University in Massachusetts and attended the Department of Philosophy at the University of Wisconsin Graduate School. Dr. Epstein received the M.D. from Rush Medical College in Chicago in 1980. He performed residencies in medicine and has a specialty certification from the American Board of Internal Medicine. He joined the NIH in 1983. He was the recipient of the Public Health Service Outstanding Service medal in 1993. Dr. Epstein’s research centers on obtaining a molecular and physiological understanding of human heart disease by studying inherited forms of cardiomyopathy.

Owen N. Witte, M.D.
Director of Eli and Edythe Broad Center of Regenerative Medicine & Stem Cell Research and Distinguished Professor of Microbiology, Immunology and Molecular Genetics at UCLA, Los Angeles, CA
http://faculty.pharmacology.ucla.edu/institution/personnel?personnel_id=45695

Lymphocyte Signal Transduction through Bruton’s Tyrosine Kinase
Dr. Owen N. Witte is professor of microbiology and immunology at the UCLA School of Medicine. Dr. Witte’s research focuses on the development of the immune response and growth regulation of hematopoietic stem cells by the abl oncogene, Bruton’s tyrosine kinase and other mechanisms. He is an investigator of the Howard Hughes Medical Institute and holds the David Saxon Presidential Chair in Developmental Immunology. Dr. Witte received the M.D. from Stanford University School of Medicine in 1976. He performed postdoctoral research with Dr. David Baltimore at M.I.T. until 1980, when he joined the faculty at UCLA. Dr. Witte has served on a number of scientific review panels and editorial boards, including the boards of Molecular and Cellular Biology, Science and Cell. He was elected to membership in the National Academy of Sciences this past year.

Philip C. Hanawalt, Ph.D.
Professor of Biology and Dermatology, Stanford University, Stanford, CA

Relevance of Transcription-Coupled DNA Repair to Human Disease
Dr. Philip C. Hanawalt is professor of biology at Stanford University. He received a Ph.D. in biophysics in 1959 from Yale University where he worked with Richard Setlow on the effects of ultraviolet light on DNA synthesis in E. coli. He performed postdoctoral work with Ole Maaløe at the University of Copenhagen and with Robert Sinsheimer at Caltech. His work on DNA repair in prokaryotes and eukaryotes set the stage for the discovery of a DNA repair defect in the human disease xeroderma pigmentosum. More recently Dr. Hanawalt and colleagues showed that a repair pathway (transcription-coupled repair) is used for transcribed strands of expressed genes. This repair mechanism operates in human cells lacking p53, whereas global DNA repair is deficient. Dr. Hanawalt serves on numerous scientific advisory boards, is on the board of reviewing editors for Science, and acts in an editorial capacity for six other journals. He is a member of the National Academy of Sciences.

David S. Bredt, M.D., Ph.D.
Global Head of Neuroscience Discovery, Johnson & Johnson, San Diego, CA

Nitric Oxide Synthase and Duchenne Muscular Dystrophy
Dr. David S. Bredt trained at the John Hopkins University School of Medicine and then served on the faculty in the Physiology Department at UC San Francisco School of Medicine. He joined Eli Lilly as head of its research on Alzheimer’s, schizophrenia and other neurological diseases in 2004. He became Global Head of Neuroscience Discovery at Johnson & Johnson in San Diego in 2011.

Roger W. Wiseman, Ph.D.
Laboratory of Molecular Carcinogenesis, National Institute of Environmental Health Sciences

Identification of BRCA1- Promises and Paradoxes
Dr. Roger W. Wiseman is senior staff fellow and leader of the Molecular Carcinogenesis Group at the National Institute of Environmental Health Sciences in Research Triangle Park, NC. He received his Ph.D. in Oncology in 1986 from the University of Wisconsin and subsequently joined NIEHS. Dr. Wiseman has been an invited speaker at many meetings including the Gordon Research Conference on Cancer, the Human Genetics Congress, and the International Conference on Carcinogenesis and Risk Assessment. In addition to his studies on BRCA1, which is linked to breast and ovarian cancer susceptibility, Dr. Wiseman studies genes involved in lung tumor induction and melanoma.

William B. Guggino, Ph.D.
Director of the Cystic Fibrosis Research Development Program, Professor/Chairman of Physiology and Professor/Vice Chairman of Pediatrics, Johns Hopkins University, Baltimore, MD
http://physiology.bs.jhmi.edu/department-member/william-guggino-chair-department-of-physiology-professor-with-secondary-appointment-in-pediatrics/

The Molecular Defect in Cystic Fibrosis: Can We Correct It?
Dr. William B. Guggino is professor of physiology and of pediatrics at the Johns Hopkins University School of Medicine where he has been since 1982. He received his Ph.D. in 1978 from the University of North Carolina at Chapel Hill and subsequently did postdoctoral studies at Yale University School of Medicine. He has served on several grant review panels and editorial boards. Dr. Guggino studies chloride channels, especially the one mutated in cystic fibrosis patients. He is trying to understand the defect found in these patients and is studying modes of correcting these defects by drug and gene therapy.

Inder M. Verma, Ph.D.
American Cancer Society Professor of Molecular Biology, Laboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA; Editor-in-Chief of PNAS
http://www.salk.edu/faculty/verma.html

Prospects in Human Gene Therapy
Dr. Inder M. Verma is professor at the Molecular Biology and Virology Laboratory of Salk Institute in La Jolla, where he has been since 1974. He received his Ph.D. in 1971 from the Weizmann Institute in Israel and subsequently did postdoctoral studies with Nobel laureate David Baltimore at MIT. He has served on several grant review panels and editorial boards and is chair of the scientific advisory board of Somatix Therapy Corporation in Alameda. Dr. Verma is well known for studies on oncogene and proto-oncogene expression and function. His studies on gene transfer using retroviral vectors provide model systems and practical approaches for employing gene therapy to treat or cure some human genetic diseases.

Henry F. Epstein, M.D.
Dr. Epstein passed away in 2013 while serving as the Cecil H. & Ida M. Green Distinguished University Chair in Neuroscience & Cell Biology, University of Texas, Galveston, TX
http://jcs.biologists.org/content/126/4/871

Recent Genetic Advances in Human Muscle Disease
Dr. Henry F. Epstein is Professor of Neurology, Cell Biology and Biochemistry at Baylor College of Medicine in Houston. He received an A.B. degree from Columbia University and the M.D. in 1968 from Stanford University. Dr. Epstein performed postdoctoral work at the National Institutes of Health and the Medical Research Council in Cambridge England. Dr. Epstein is well known as a muscle cell biologist, geneticist and biochemist and has published extensively on myofibril structure and assembly in the genetically manipulatable nematode Caenorhabditis elegans. In addition he has turned his attention to defining the genetic defects associated with human muscle diseases including myotonic muscular dystrophy and hypertrophic cardiomyopathy. Dr. Epstein has served as co-director of the Jerry Lewis Neuromuscular Disease Research Center at Baylor and is a member of the Scientific Advisory Committee for the Muscular Dystrophy Association.

Savio Woo, Ph.D.
Professor of Medicine, Hematology, and Medical Oncology; Genetics and Oncological Sciences, Icahn School of Medicine, Mt. Sinai Hospital, New York, NY
http://www.acgtfoundation.org/leadership/savio-l-c-woo/

Molecular Genetics of PKU: From Anthropology to Gene Therapy
Dr. Savio L.C. Woo is a Professor in the Department of Cell Biology at Baylor College of Medicine and an Investigator of the Howard Hughes Medical Institute. He is founding Director of the Center for Gene Therapy at Baylor College of Medicine. Dr. Woo received a B.S. from Loyola College and a Ph.D. from the University of Washington.

Charles D. Laird, Ph.D.
Professor Emeritus of Biology
Associate Director for Fragile X Research, University of Washington’s Center on Human Development and Disability, Seattle, WA
http://depts.washington.edu/lairdlab/people.htm

Applying Principles of Drosophila Chromosomes to the Human Genetic Disorder, the Fragile-X Syndrome
Dr. Charles D. Laird, Professor of Zoology and Adjunct Professor of Genetics at the University of Washington received his B.S. from the University of Oregon in Eugene and obtained his Ph.D. from Stanford University in 1966. He then pursued postdoctoral studies at the University of Washington prior to his appointment to the faculty at the University of Texas at Austin. In 1971 he moved to the University of Washington. Dr. Laird is well known for his studies on the structure and replication of insect chromosomes. Recently he has turned his attention to studying the fragile-X syndrome in humans. He has co-authored a number of papers in this area and presented seminars at several international meetings on human genetic diseases. His research is supported by grants from the NSF, NIH and the Joseph P. Kennedy, Jr. Foundation.

Francis S. Collins, M.D., Ph.D.
Director of the National Institutes of Health
http://www.nih.gov/about/director/directorbio.htm

Progress in the Search for the Cystic Fibrosis Gene
We are fortunate to have Dr. Francis Collins from the University of Michigan Medical Center to present his latest results regarding the search for the cystic fibrosis gene. Dr. Collins is Chief of the Division of Medical Genetics and an Associate Investigator of the Howard Hughes Medical Institute. He received his un­dergraduate training at the University of Virginia and went on to receive the M.S. and Ph.D. degrees from Yale. He then obtained an M.D. from the University of North Carolina School of Medicine in 1977. In addition to working on cystic fibrosis, Dr. Collins is studying neurofibromatosis, Huntington’s disease and the globin gene. Dr. Collins has received numerous awards and honors and has published in the top journals. He cur­rently has three research grants from the National Institutes of Health as well as funding from the Heredi­tary Disease Foundation, the March of Dimes and the Cystic Fibrosis Foundation.