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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Appendix C

Speaker Biographies

Steve Brown earned his Ph.D. at Cambridge University, and before he joined the Medical Research Council, he was professor of genetics at Imperial College, London. His research interests cover mouse functional genomics, including the use of mouse mutagenesis and comparative genomic analysis to study the genetic basis of disease and to develop pre-clinical disease models. A particular focus has been the use of mouse models to study the molecular basis of genetic deafness. Along with Karen Steel, he discovered myosin VIIA as the gene underlying the shaker1 mutant, one of the first deafness genes to be identified. Subsequently, he has developed interests in the protein complexes that are involved with stereocilia elongation in hair cells in the inner ear. In addition, over the last 10 years he has led a substantial research effort in the genetics of otitis media or glue ear, a common cause of hearing loss in children, employing mouse models to elaborate the key genetic pathways involved and develop novel therapeutic strategies. He has served on numerous advisory boards, and his current appointments include the Advisory Council for the National BioResource Centre, Japan; Science Advisory Board (SAB) for the Helmholtz Centrum, München; SAB for the Centre Intégratif de Génomique (CIG), University of Lausanne; and the Strategic Policy Committee, Institut de Génétique et de Biologie Moléculaire et Cellulaire, Strasbourg. He is the current chair of the International Mouse Phenotyping Consortium Steering Committee.

Robert Burgess’s lab seeks to understand the molecular mechanisms of synapse formation and maintenance at two sites in the nervous system: the peripheral neuromuscular junction and the retina. In all of these studies,

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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the Burgess lab is addressing basic molecular mechanisms, but these basic mechanisms have relevance to human neuromuscular and neurodevelopmental disorders. The lab’s continued research on the genetics underlying these disorders, and the continuing effort to identify new genes involved in these processes, will increase our understanding of the molecules required to form and maintain synaptic connectivity in the nervous system. Dr. Burgess received his B.S. in biochemistry from Michigan State University, and his Ph.D. in neuroscience from Stanford University. After doing postdoctoral research at Washington University, St. Louis, he joined the faculty of The Jackson Laboratory in Bar Harbor, Maine, in 2001. Dr. Burgess’s research is funded by the Muscular Dystrophy Association and the National Institutes of Health. Among other leadership positions, he is the principal investigator of The Jackson Center for Precision Genetics.

Paul Burridge is an assistant professor in the Department of Pharmacology at Northwestern University Feinberg School of Medicine and founding faculty of the Center for Pharmacogenomics. Dr. Burridge began his career in genomics and bioinformatics at the Sanger Institute working on the human and mouse genome projects. He completed a Ph.D. in Human Stem Cell Biology at the University of Nottingham before pursuing postdoctoral fellowships at the Johns Hopkins University Institute for Cell Engineering in Pediatric Oncology and then at Stanford University Institute for Stem Cell Biology and Regenerative Medicine. Dr. Burridge became an instructor in cardiovascular medicine at Stanford in 2013 before joining Northwestern in 2015. For more than a decade, he has worked on the applications of human induced pluripotent stem cells disease modeling, specifically the pharmacogenomic and molecular mechanisms of chemotherapy-induced cardiomyopathy and heart failure. Dr. Burridge is the recipient of the National Institutes of Health National Heart, Lung, and Blood Institute Pathway to Independence Award and a Dixon Young Investigator Award, and in 2016 he was elected a fellow of the American Heart Association.

John (Seán) Clohessy is director of the Mouse Hospital/Preclinical Murine Pharmacogenetics Facility at the Beth Israel Deaconess Medical Center and Harvard Medical School (BIDMC/HMS) in Boston, Massachusetts. Dr. Clohessy received his Ph.D. in 2005 from University College London, where he studied the role of BCL2 family proteins in the regulation of apoptosis and leukemia. Following this, he completed post-graduate training at Memorial Sloan Kettering Cancer Center and then at the Beth Israel Deaconess Medical School/Harvard Medical School, where he is currently instructor in medicine. His current focus as director of the Mouse Hospital at BIDMC/HMS focuses on the development of best practices approaches for implementation of pre- and co-clinical trials in mice, with the aim to

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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develop a “Mouse Hospital” whereby genetically engineered mouse models that faithfully model human disease can be treated in a manner similar to how they would be treated in human patients. In particular, he is interested in the development and utilization of genetically engineered mouse models for pre- and co-clinical applications to facilitate genetic stratification of patients for focused and rapid clinical evaluation of novel therapies and therapeutic strategies in the treatment of cancer. In addition, Dr. Clohessy has an active research interest in the role of the ribosome and cellular translation in disease.

Genya Dana joins the Forum from the U.S. Department of State where she spent 5.5 years working at the nexus of science and foreign policy in the Office of the Science and Technology Adviser to the Secretary of State. She is an internationally recognized expert on the policy and science of synthetic biology and other emerging biotechnologies, and she led the development of the U.S. position and outreach on synthetic biology and gene editing at the international level. She served as a negotiator on science, technology, and innovation in multilateral organizations like the United Nations, worked to advance science, technology, and innovation for sustainable development with a focus on Africa, and developed international partnerships to support scientific research on brain science and biological engineering. Dr. Dana grew up on a small family farm in Mississippi, completed a bachelor’s degree in biology from Emory University in Atlanta, and spent several years working in finance before returning to graduate school to combine science and policy. She has an M.Sc. in science, technology, and environmental policy and a Ph.D. in ecological risk assessment, both from the University of Minnesota.

Mary Dickinson is an internationally recognized expert in imaging, genetics, bioengineering, and vascular biology with more than 100 peer-reviewed publications. Her work involves the development of novel imaging strategies to define the genetic and environmental causes of congenital birth defects and understand mechanisms underlying angiogenesis and vascular remodeling. In addition to Dr. Dickinson’s research efforts, she serves as the director of the Optical Imaging and Vital Microscopy core, as an executive member of the Cardiovascular Research Institute, and as a principal investigator for the National Institutes of Health Knockout Mouse Project. Dr. Dickinson is also a fellow of the American Institute of Medical and Biological Engineers and recently became the associate dean of research at Baylor College of Medicine.

Ulrich Dirnagl’s research focuses on stroke, cerebral blood flow regulation, and brain imaging. In preclinical models, as well as clinical trials, he and his

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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coworkers and collaborators explore mechanisms by which brain ischemia leads to cell death, and develop novel methods to intercept mechanisms of damage in acute brain damage, as well as to foster regeneration and repair of the lesions. Dr. Dirnagl is particulary interested in how the brain protects itself (“endogenous neuroprotection”), and how the brain interacts with other systems of the body after it has been injured. Closely linked to his interest in stroke pathophysiology is his interest in the coupling of regional blood flow to neuronal acitivity, the mechanism underlying functional brain imaging with magnetic resonance (MR) and positron-emission tomography. Beyond imaging structure and function of the central nervous system he and his team are developing, validating and using techniques that allow the non-invasive imaging of brain biochemistry and molecular signaling. To this end they use optical, MR, and nuclear medicine approaches in mice and humans. To improve the predictiveness of preclinical translational research he is actively promoting the introduction of quality standards for experimental design and reporting, as well as international collaboration in large, phase III-type preclinical trials. Through meta-research he is trying to identify opportunities for improving research practice and to obtain evidence for the impact of targeted interventions. At the Charité Universitätsmedizin Berlin Dr. Dirnagl serves as director of the Department of Experimental Neurology. Since 2017 he is also the founding director of the Center for Transforming Biomedical Research (CTBR) at the Berlin Institute of Health. CTBR aims at overcoming the roadblocks in translational medicine by increasing the value and impact of biomedical research through maximizing the quality, reproducibility, generalizability, and validity of research.

Brian Fiske is the senior vice president for research programs at The Michael J. Fox Foundation (MJFF) for Parkinson’s Research. Dr. Fiske co-manages a team of professionals who stay closely linked to the Parkinson’s community in order to develop an aggressive and innovative agenda for accelerating research and drug development for Parkinson’s disease. This ensures that MJFF priorities reflect and best serve the ultimate needs of patients. Dr. Fiske regularly meets with academic and industry scientists around the world to identify promising ideas to support, providing troubleshooting and ongoing management of projects as they go forward. He currently oversees the teams focused on MJFF’s strategies for developing disease-modifying and symptomatic therapies for Parkinson’s patients. Dr. Fiske earned an undergraduate degree in biology from Texas A&M University and a Ph.D. in Neuroscience from the University of Virginia. After completing postdoctoral research at Columbia University, Dr. Fiske spent several years as an editor for the prestigious scientific journal, Nature Neu

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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roscience. He brings this broad experience and knowledge to the Foundation to help bring new treatments to people with Parkinson’s.

Alison Harrill is a geneticist at the National Institutes of Health/National Institute for Environmental Health Sciences in the Division of the National Toxicology Program (NTP). Her research interests include investigating the role of genetic sequence variation and population dynamics as it relates to toxicity susceptibility for the purposes of risk management. In addition, she has used rodent population models, like the Diversity Outbred mice, to investigate differential modes of action of toxicity susceptibility using toxicogenomic investigations in resistant and susceptible individuals. At the NTP, she is currently working toward qualification of cell-based screens to assessing potency of developmental neurotoxic agents using mouse population models in order to better extrapolate risk to genetically diverse human populations. She currently serves as an associate editor for Toxicological Sciences, co-chair of the Health and Environmental Sciences Institute’s (HESI’s) Application of Genomics to Mechanism-Based Risk Assessment Technical Committee, co-chair of the HESI miRNA Biomarkers Working Group, co-chair of the Society of Toxicology, Contemporary Concepts in Toxicology Committee, and past secretary/treasurer of the Toxicology Division within the American Society for Pharmacology and Experimental Therapeutics. Dr. Harrill received her Ph.D. in Toxicology from the University of North Carolina at Chapel Hill (2009). Dr. Harrill and her research team have received several awards, including the Burroughs Wellcome Fund Award for Innovation in Regulatory Science (2013-2016), the Society of Toxicology Outstanding Published Paper in Advancing the Science of Risk Assessment Award (2009), and the Society of Toxicology Best Paper Published in Toxicological Sciences (2016).

Elizabeth Heitman is a professor in the Program in Ethics in Science and Medicine at the University of Texas Southwestern Medical Center in Dallas, Texas. Her work focuses on cultural issues and international aspects of ethics in medicine, biomedical science, and public health; her research examines international standards of research ethics, education in the responsible conduct of research, and trainees’ awareness of professional and cultural norms. She is co-director of the research ethics education program “Formação Colaborativa na Ética em Pesquisa (Collaborative Research Ethics Education),” sponsored by the National Institutes of Health Fogarty International Center, with colleagues from Vanderbilt University Medical Center in Nashville, Tennessee, and the Universidade Eduardo Mondlane in Maputo, Mozambique. Dr. Heitman previously directed a similar program with the Hospital Nacional de Niños in San José, Costa Rica, and was principal investigator of the National Science Foundation–funded study

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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“Research Integrity in the Education of International Science Trainees.” She is a member of the Board on Life Sciences’ Standing Committee on Educational Institutes for Teaching Responsible Science. Through the National Academies, Dr. Heitman has taught in international faculty development projects on responsible science in the Middle East and North Africa, as well as Malaysia and Indonesia. She previously chaired the National Academies Committee on the Elaboration of a National Curriculum in Bioethics and Responsible Conduct of Science for Algeria, advising the Algerian Ministry of Higher Education, and recently served as co-chair of the Committee on Gene Drive Research in Non-Human Organisms: Recommendations for Responsible Conduct. Dr. Heitman received her Ph.D. in Religious Studies in 1988 from Rice University’s joint program in biomedical ethics with the University of Texas–Houston Health Science Center.

Merel Ritskes Hoitinga is a professor in evidence-based laboratory animal science at the Department for Health Evidence at the Radboud University Medical Center, Nijmegen, the Netherlands. She founded SYRCLE (www.syrcle.nl) in 2012, focusing on education, coaching, and research in the field of systematic reviews of animal studies. Systematic reviews lead to the actual implementation of the 3Rs, a more evidence-based choice of animal models and transparency in quality and translation of animal studies. In 2017 SYRCLE won the Cochrane REWARD 2nd prize, illustrating the importance of this work for Cochrane and the Lancet REWARD Alliance campaign (increasing value and reducing waste in research). SYRCLE participates in international networks: CAMARADES, Evidence-Based Toxicology, Evidence-Based Research Network, GRADE and Evidence Synthesis International, and is preparing an application for a Cochrane methods group. From 1997 to 2005 Dr. Ritskes Hoitinga was a professor in laboratory animal science and comparative medicine at the Faculty of Health Sciences, University of Southern Denmark. In 2017 she was a visiting professor at the Department for Clinical Medicine at Aarhus University.

India Hook-Barnard is director of research strategy and associate director, precision medicine at the University of California, San Francisco (UCSF). She serves as director of the UCSF Precision Medicine Platform Committee, which aims to conceptualize and build precision medicine as a central overarching institutional vision at UCSF. She helped launch and serves as executive director for the California Initiative to Advance Precision Medicine. In the UCSF Office of Science Policy and Strategy, she works with scientific experts and leaders from across academia, industry, and government sectors to identify and frame scientific opportunities, technical feasibility, and organizational challenges. She develops and supports efforts to secure major research and infrastructure funding, supports research train-

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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ing programs, and provides advice, consultation, and analysis on scientific initiatives. Dr. Hook-Barnard’s primary interests are in areas of emerging science, technology, and medicine, spanning fundamental discovery to translational application, clinical practice, and population health research, including issues of policy, regulation, ethical and societal concerns, and workforce. From 2008 to 2015, she worked as a senior program officer with the National Academy of Sciences (NAS) and the Institute of Medicine (IOM). At the NAS and the IOM, she developed and administered a variety of programs, working with academic faculty, scientists, engineers, clinicians, business leaders, and policy experts. She served as the study director for six National Academies reports, including Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease (2011), Determining Core Capabilities in Chemical and Biological Defense Science and Technology (2012), and Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk (2015). Dr. Hook-Barnard was a postdoctoral research fellow at the National Institutes of Health. She earned her Ph.D. in microbiology-medicine from the Department of Molecular Microbiology and Immunology at the University of Missouri.

Nicholas (Nico) Katsanis obtained his baccalaureate degree in genetics from the University College London in 1993 and his doctorate degree from Imperial College London in 1997. While at Imperial College, he worked with Dr. Elizabeth Fisher on the genetics of Down syndrome. He completed his postdoctoral work in the laboratory of Dr. James Lupski within the Department of Molecular and Human Genectis at Baylor College of Medicine in Houston and transitioned his studies to Bardet-Biedl syndrome. In 2002, he established his independent research lab at the Institute of Genetic Medicine, Johns Hopkins University, where he led studies that unified several allied conditions under the ciliopathy umbrella. Dr. Katsanis demonstrated that Bardet-Biedl syndrome is caused by centrosomal/basal body dysfunction, establishing that dysfunction at the primary cilium can give rise to a large group of disorders with both clinical and genetic overlap (the ciliopathy model). In 2009, he moved to Duke University to establish the Center for Human Disease Modeling (CHDM) and is the director. He developed a structure that aims to facilitate collaboration across disciplines and to develop physiologically relevant tools to study variation found in human patient genomes. As part of that effort, Dr. Katsanis leads the Taskforce for Neonatal Genomics. This multidisciplinary group of physicians and basic scientists strives to synthesize genomic and biological data for the faster diagnosis, improved/focused clinical care, and potential therapeutic paradigms for infants and neonates with genetic conditions. In parallel, the CHDM pursues questions centered on the signaling roles of vertebrate cilia, the translation of signaling pathway defects on the causality

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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and possible treatment of ciliary disorders, and the dissection of second-site modification phenomena as a consequence of genetic load in a functional system. In recognition of his work, Dr. Katsanis was awarded the Young Investigator Award from the American Society of Nephrology in 2009, the E. Mead Johnson Award from the Society for Pediatric Research in 2012, and the Curt Stern Award from the American Society of Human Genetics in 2017 and has delivered several distinguished lectures. Dr. Katsanis is a professor in the Departments of Cell Biology and Pediatrics and holds the Jean and George Brumley Distinguished Professorship.

Jonathan Kimmelman is associate professor at McGill University in the Biomedical Ethics Unit/Social Studies of Medicine, with a cross appointment in Experimental Medicine. His research centers on ethical, policy, and scientific dimensions of drug and diagnostics development, and he founded and directs the Studies of Translation, Ethics and Medicine. In addition to his book, Gene Transfer and the Ethics of First-in-Human Experiments (Cambridge Press, 2010), major publications have appeared in Science, JAMA, BMJ, and Hastings Center Report. Dr. Kimmelman received the Maud Menten New Investigator Prize (2006), a Canadian Institutes for Health Research (CIHR) New Investigator Award (2008), and a Humboldt Bessel Award (2014). He has served on various advisory bodies within the National Heart, Lung, and Blood Institute, the U.S. National Academy of Medicine, and the CIHR, and makes frequent appearances in the news media. He chaired the International Society of Stem Cell Research Guidelines for Stem Cell Research and Clinical Translation revision task force 2015-2016.

Kenjiro Kosaki received clinical and research training in medical genetics at the University of California, San Diego, and Baylor College of Medicine and received board certification in clinical genetics from the American Board of Medical Genetics. Currently, he is serving as the co-principal investigator for two government-funded national research projects of Japan in the field of medical genomics and rare diseases. He was the organizer of the Fourth International Conference on Rare and Undiagnosed Diseases held in Tokyo in November 2016.

Jens H. Kuhn is a principal at Tunnell Government Services (TGS), Inc., Bethesda, Maryland, tasked as the lead virologist (contractor) at the Integrated Research Facility (IRF). He is also TGS team leader for all IRF-Frederick TGS contractors. Dr. Kuhn specializes in highly virulent viral pathogens. He is the author of Filoviruses: A Compendium of 40 Years of Epidemiological, Clinical, and Laboratory Studies (Vienna: Springer, 2008) and co-author of The Soviet Biological Weapons Program—A History

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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(Cambridge: Harvard University Press, 2012) and has studied and worked in Germany, Italy, Malta, Russia, South Africa, and South Korea. In the United States, he rotated through or worked at Harvard Medical School, Boston, Massachusetts; the Arthropod-Borne Infectious Disease Laboratory in Fort Collins, Colorado; the Centers for Disease Control and Prevention in Atlanta, Georgia; and the U.S. Army Medical Research Institute of Infectious Diseases in Frederick, Maryland. Dr. Kuhn was the first Western scientist with permission to work in the former Soviet biological warfare facility SRCVB “Vector” in Siberia, Russia, within the U.S. Department of Defense Cooperative Threat Reduction Program. Dr. Kuhn was a contributor to the Center for International and Security Studies at Maryland’s Controlling Dangerous Pathogens Project and a member of the Center for Arms Control and Nonproliferation’s Scientists Working Group on Chemical/Biological Weapons. He is currently chairing the International Committee on Taxonomy of Viruses Filoviridae and Mononegavirales Study Groups and is a subject-matter expert for the National Center for Biotechnology Information for all mononegaviruses; is a member of the editorial boards of Applied Biosafety—Journal of the American Biological Safety Association, Archives of Virology, BioMed Research International, Journal of Bioterrorism and Biodefense, PLoS ONE, PLoS Pathogens, Viruses, Virologica Sinica, Voprosy Virusologii, and World Journal of Virology; was a member of the U.S. National Academy of Sciences’ committee on animal models for assessing countermeasures to bioterrorism agents; and is continuously involved with the American Association for the Advancement of Science and the U.S. Department of State bioengagement efforts in the Broader Middle East and North Africa region, Turkey, and the Newly Independent States.

Amy K. LeBlanc is the director of the intramural National Cancer Institute’s Comparative Oncology Program. In this position she conducts preclinical mouse and translational canine studies that are designed to inform the drug and imaging agent development path for human cancer patients. She also advises leading pharmaceutical companies as well as the National Cancer Institute’s (NCI’s) Division of Cancer Treatment and Diagnosis on the inclusion of pet dogs with cancer into the development path of novel approaches for a variety of malignancies, including immunotherapeutics, targeted small molecules, oncolytic viruses, and cancer imaging agents. She directly oversees the NCI Comparative Oncology Trials Consortium, which provides infrastructure necessary to connect participating veterinary academic institutions with stakeholders in drug development to execute fit-for-purpose comparative clinical trials in novel therapeutics and imaging agents. Prior to her appointment at the National Institutes of Health, Dr. LeBlanc was an associate professor with tenure and director of translational research at the University of Tennessee College of Veterinary Medi-

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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cine and Graduate School of Medicine. Dr. LeBlanc’s group at the University of Tennessee published the first comprehensive studies describing molecular imaging of dogs and cats using positron-emission tomography/computed tomography, focusing on the forward and back-translation of 18F-labeled radiopharmaceuticals.

Jennifer Maier is a postdoctoral research associate at the University of California, Los Angeles, in the lab of Karen Sears. Prior to moving to UCLA, she was in the Sears lab at University of Illinois at Urbana-Champaign. Her Ph.D. work was done at the University of Florida under Brian D. Harfe, where she focused on the development of the intervertebral disc using a variety of transgenic and mouse models. Her interests are broadly in evolutionary developmental (evo-devo) biology and using non-classical mammalian models to study diversity. She has worked on a number of projects in the Sears lab, including investigating mammalian limb diversity, mammalian digit reduction, and teratogenesis. Currently she is characterizing the spermatogonial stem cells of the gray short-tailed opossum (Monodelphis domestica) and developing methods to generate the first transgenic marsupials.

Arjun (Raj) Manrai is an instructor at the Department of Biomedical Informatics at Harvard Medical School. Dr. Manrai received an undergraduate degree in physics from Harvard College and completed his Ph.D. in bioinformatics and integrative genomics at the Harvard-Massachusetts Institute of Technology Division of Health Sciences and Technology. Dr. Manrai works on machine learning and statistical methods to improve the use of genomic and laboratory data in the clinic. His work has been published in the New England Journal of Medicine and the Journal of the American Medical Association, and featured in The Wall Street Journal and The New York Times and on National Public Radio.

Keith Mansfield is director of Discovery and Investigative Pathology at Novartis Institutes for Biomedical Research and heads a molecular pathology laboratory in Cambridge, Massachusetts. Prior to joining Novartis, Dr. Mansfield was an associate professor of pathology at Harvard Medical School. He was principal investigator of a number of National Institutes of Health (NIH) grants funded through the National Institute of Allergy and Infectious Diseases, National Institute of Aging, and National Institute of Diabetes and Digestive and Kidney Diseases and was a permanent member and chair of the NIH Comparative Medicine Review Committee. During this time he was director of a T32 NIH training grant in comparative pathology and mentored a number of postgraduate veterinarians. His research was focused on the experimental pathology of infectious diseases,

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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and he is author/co-author on more than 200 peer-reviewed research publications. Since joining Novartis in 2011, he has led a molecular pathology laboratory utilizing precision models in drug discovery and development. In 2016 he received a Novartis outstanding scientist award for his work. He obtained his bachelor’s and D.V.M. degrees from Cornell University and is currently an adjunct professor at Cornell University, School of Veterinary Medicine. He trained in veterinary pathology at Harvard Medical School and is board certified in veterinary pathology by the American College of Veterinary Pathologists.

Megan McCain is an assistant professor and the Chonette Early Career Chair in the Department of Biomedical Engineering and the Department of Stem Cell Biology and Regenerative Medicine at the University of Southern California (USC). Dr. McCain earned her B.S. in biomedical engineering at Washington University in St. Louis and her Ph.D. in engineering sciences at Harvard University. As a graduate student, she engineered cardiac cells and tissues to investigate mechanotransduction in the context of cardiac development and disease. She continued as a postdoctoral researcher at the Wyss Institute for Biologically Inspired Engineering at Harvard University, where she engineered microscale human cardiac tissues on platforms with integrated functional assays, known as “heart-on-a-chip,” for patient- specific disease modeling and medium-throughput drug screening. In 2014, Dr. McCain joined USC and established the Laboratory for Living Systems Engineering to engineer and implement novel “organ-on-chip” platforms for human disease modeling and drug screening, with a focus on cardiac and skeletal muscle. Dr. McCain is a recipient of a Scientist Development Grant from the American Heart Association and a Starter Grant from the Amyotrophic Lateral Sclerosis Association. She has also been recognized as a Top Innovator Under 35 by the Massachusetts Institute of Technology Technology Review and a Rising Star by the Cellular and Molecular Bioengineering sub-group of the Biomedical Engineering Society.

Catherine Nguyen has worked since 1984 in experimental biology, mainly in molecular biology. In 1991 Dr. Nguyen became interested in gene expression analysis using large-scale approaches to question human diseases. She currently serves as the director of the ITMO Genetique in Paris, France. Dr. Nguyen received her Ph.D. in immunology from the University of AixMarseille II.

Daryl Pritchard is the senior vice president of science policy at the Personalized Medicine Coalition (PMC), where he leads PMC efforts to increase awareness and understanding of personalized medicine; identify and address barriers to the adoption of personalized medicine into the healthcare system;

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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and develop and promote appropriate clinical, healthcare infrastructure, regulatory, and payment policies. Before coming to PMC, Dr. Pritchard served as the director of policy research at the National Pharmaceutical Council (NPC), where he led NPC’s policy research, alliance development, and government relations efforts in the areas of personalized medicine, the heterogeneity of treatment effects, and recognition of the value of specialty biopharmaceuticals. Prior to joining the NPC, Dr. Pritchard served as the director of Research Programs Advocacy and Personalized Medicine at the Biotechnology Industry Organization, where he developed and expanded the organization’s diagnostics and personalized medicine policy program. He also spent 3 years as the director of government affairs for the American Association for Dental Research, and the American Dental Education Association. Dr. Pritchard received his Ph.D. and master’s degree in genetics from The George Washington University, and completed a postdoctoral research fellowship at the Children’s National Medical Center. He was awarded the first American Society of Human Genetics/National Human Genome Research Institute Fellowship in Genetics and Public Policy, where he worked as a health legislative assistant in the House of Representatives.

Richard Sharp is the director of the Biomedical Ethics Program at the Mayo Clinic. Prior to joining Mayo Clinic in July 2013, he was director of bioethics research at Cleveland Clinic and co-director of the Center for Genetic Research Ethics and Law at Case Western Reserve University, one of six National Institutes of Health Centers of Excellence in Ethics Research. Dr. Sharp has published widely on topics in biomedical ethics, including clinical ethics consultation, informed consent, financial conflicts of interest, and ethical tensions in patient advocacy. His current research is examining how patients and healthcare providers view new forms of personalized medicine and clinical interventions enabled by molecular diagnosis. Dr. Sharp frequently advises healthcare organizations on ethical issues and has served on advisory committees for the National Institutes of Health, Institute of Medicine, American College of Medical Genetics, and U.S. Environmental Protection Agency.

Damian Smedley is director of genomic interpretation for Genomics England, overseeing the clinical analysis of the rare disease and cancer genomes emerging from the National Health System’s 100,000 Genomes Project. He is also a senior lecturer at Queen Mary University London, where his research focuses on the use of phenotype data to obtain novel insights into disease causes and mechanisms. His team is involved in translational aspects for a number of projects, such as the International Mouse Phenotyping Consortium. In collaboration with other members of the Monarch Initiative he has developed tools that utilize phenotype comparisons for

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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candidate gene prioritization, particularly for next-gen sequence interpretation of rare disease patients, as in the Exomiser software suite.

James Stevens is a distinguished research fellow at Lilly Research Laboratories and is recognized internationally as an expert in drug safety science and the application of systems biology in safety testing. For over 4 decades in government, academics, and industry he has studied molecular and cellular responses to the metabolism and toxicity of drugs and xenobiotics. His current research focuses on application of systems biology to elucidate the molecular basis of pathogenesis using network-based approaches. Dr. Stevens received his Ph.D. in pharmacology from the University of Minnesota in 1980 and did postdoctoral training at the National Institutes of Health (NIH) as a National Institute of General Medical Science-Pharmacology Research Associate Training Fellow. Prior to joining Lilly Research Laboratory in 2000, he held positions at NIH, the U.S. Food and Drug Administration, the University of Vermont, and the W. Alton Jones Cell Science Center, where he was executive director. He has served on a variety of national advisory committees, including the Health and Environmental Sciences Institute Board of Trustees, National Advisory General Medical Sciences at NIH, National Toxicology Program Board of Scientific Councilors, the Environmental Protection Agency Subcommittee on Chemical Safety for Sustainability, as well as the boards of directors for Argonex Pharmaceuticals and Upstate Biotechnology. As a member of the Translational Safety Strategic Governance Group for the Innovative Medicine Initiative he was instrumental in launching the Translational Quantitative Systems Toxicology project in January 2017, a 5-year project including more than 30 pharmaceutical and academic partners. He received the Achievement Award from the Society of Toxicology in 1994 and was elected a fellow of the American Association for the Advancement of Sciences in 1996.

D. Lansing Taylor’s laboratory has embraced the Quantitative Systems Biology approach to drug discovery and development that integrates experimental and computational methods, especially in metastatic breast cancer, neurodegenerative diseases, and liver diseases. Dr. Taylor began his academic career at Harvard University and remained at Harvard until 1982, developing and using novel fluorescence-based reagents and imaging technologies to investigate fundamental cellular processes in living cells. He then moved to Carnegie Mellon University (CMU) as a professor of biological sciences and as director of the Center for Fluorescence Research in the Biomedical Sciences continuing to develop and to apply novel fluorescence-based technologies in biology and medicine. Dr. Taylor co-founded Biological Detection Systems in the early 1990s to commercialize research light microscope imaging systems and the multi-color cyanine

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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dyes for fluorescence detection in the life sciences, which is now part of General Electric Life Sciences. Dr. Taylor left CMU in 1996 to start and lead a series of companies: Cellomics-High Content Screening, now part of ThermoFisher; Cellumen-early safety assessment, now part of Cyprotex, and finally a private company, Cernostics-cancer diagnostics. Dr. Taylor returned to academia at the end of 2010 to continue his academic interests, which now link large-scale cell and tissue profiling with computational and systems biology to optimize drug discovery and diagnostics. In addition, Dr. Taylor has collaborated with investigators in the University of Pittsburgh Drug Discovery Institute to develop methods to detect and to quantify biologically relevant heterogeneity in phenotypic assays and in pathology tissue sections. He has also led a team that has developed human organs on chips, starting with the liver, in order to explore acute and chronic toxicity, as well as to create long-term human models of disease.

Kristina Thayer has served as the Integrated Risk Information System (IRIS) division director, located within the U.S. Environmental Protection Agency (EPA) National Center for Environmental Assessment since January 2017. IRIS assessments identify the potential for a chemical to cause cancer or non-cancer health effects in people and are considered the top tier source of toxicity information used by EPA and other agencies to inform national standards, clean-up levels at local sites, and set advisory levels. IRIS uses systematic review methods to evaluate epidemiological and toxicological literature and include consideration of relevant mechanistic evidence. Prior to joining EPA, Dr. Thayer was deputy division director of analysis at the National Toxicology Program (NTP) and director of the NTP Office of Health Assessment and Translation (OHAT) located on the campus of the National Institute for Environmental Health Sciences (NIEHS). As deputy division director of analysis, she oversaw OHAT and the NTP Office of the Report on Carcinogens. She has also worked in the NTP Office of Liaison, Policy, and Review, the NIEHS Office of Risk Assessment Research, and the NTP Center for the Evaluation or Risks to Human Reproduction. Prior to joining the NTP/NIEHS, she was a senior scientist at the World Wildlife Fund and then at the Environmental Working Group. She is considered an expert on the application of systematic review methods to environmental health topics and the use of specialized software and automation approaches to facilitate conducting reviews.

David Valle is the Henry J. Knott Professor and Director of the McKusick-Nathans Institute of Genetic Medicine with a co-primary appointment in the Department of Pediatrics and joint appointments in the Departments of Molecular Biology & Genetics and Ophthalmology at Johns Hopkins University School of Medicine. His research interests include human ge-

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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netic disease with an emphasis on inborn errors of metabolism, inherited retinal degenerations, rare Mendelian disorders, and identification and understanding of genetic factors that contribute increase susceptibility for neuropsychiatric diseases, such as schizophrenia. Dr. Valle is a member of the Institute of Medicine of the National Academy of Science, Association of American Physicians, and a fellow of the American Association for the Advancement of Science. He is a past president of the American Society of Human Genetics and is the senior editor of The Metabolic and Molecular Bases of Inherited Disease. He is also director of the Predoctoral Training Program in Human Genetics and the Johns Hopkins Center for Inherited Disease Research as well as co-director of the Short Course in Medical and Experimental Mammalian Genetics at The Jackson Laboratory. In 2003 Dr. Valle received the Colonel Harland Sanders Award for Lifetime Achievement in Genetics Research and Education from the March of Dimes Foundation, the 2014 Victor A. McKusick Leadership Award, and, in 2016, the Arno Motulsky–Barton Childs Award for Excellence in Human Genetics Education, both from the American Society of Human Genetics. He was director of the Pediatric Genetics Clinic of The Johns Hopkins Hospital from 1976 to 1989, past president of The Society of Inherited Metabolic Disorders, and member and chair of the National Institutes of Health Study Sections in Mammalian Genetics and Genetic Basis of Disease. He served on the Board of Scientific Overseers of the Jackson Laboratory and the Advisory Council of the National Human Genome Research Institute. He has been a member of the editorial boards of The American Journal of Human Genetics, Human Molecular Genetics, Human Mutation, European Journal of Human Genetics, Annual Review of Nutrition, and Annual Review of Genomics and Human Genetics. Dr. Valle earned his B.S and M.D. from Duke University. He completed his pediatric residency on the Harriet Lane Service at The Johns Hopkins Hospital. He is a diplomate of the American Board of Pediatrics and the American Board of Medical Genetics, and has published more than 250 papers in reviewed journals and contributed close to 40 book chapters.

John Wikswo is the Gordon A. Cain University Professor, the A. B. Learned Professor of Living State Physics, and professor of biomedical engineering, molecular physiology and biophysics, and physics at Vanderbilt University, and the founding director of the Vanderbilt Institute for Integrative Biosystems Research and Education (VIIBRE). Trained as a physicist, he received his B.A. degree from the University of Virginia and his Ph.D. from Stanford University. He has been a member of the Vanderbilt faculty since 1977. His research has included superconducting magnetometry; the measurement and modeling of cardiac, neural, and gastric electric and magnetic fields; and non-destructive testing of aging aircraft. Since 2001,

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×

VIIBRE has developed a breadth of optical instruments and software for studying how living cells interact with each other and their environment and respond to drugs, chemical and biological agents, and other toxins, thereby providing insights into systems biology, physiology, medicine, and toxicology. For the past 5 years, Dr. Wikswo has been guiding VIIBRE engineers and scientists in the development of microfabricated organ-on-chip bioreactors that include an National Center for Advancing Translational Sciences (NCATS)-funded neurovascular unit/blood–brain barrier and the associated perfusion controllers, microclinical analyzers, and other devices that are required to integrate multiple organs-on-chips to create an in vitro micro-homunculus. His group’s most recent innovations include the ongoing development of 96-channel microformulator, funded by AstraZeneca, and a smart well plate for organs-on-chips and high-throughput biology, funded by NCATS Small Business Innovation Research Program to CFD Research Corporation. VIIBRE is also the home of the Systems Biology and Bioengineering Undergraduate Research Experience, a year-round, multiyear program funded by Gideon Searle. Dr. Wikswo has published more than 200 peer-reviewed papers, is a fellow of seven professional societies, holds 24 patents (several of which have been licensed), has multiple patents pending, and nurtures innovation among his trainees. He loves teaching and learning and sharing his enthusiasm for research and inventing with high school students, undergraduates, and graduate students.

Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×

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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
×
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Suggested Citation:"Appendix C: Speaker Biographies." National Academies of Sciences, Engineering, and Medicine. 2018. Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/25002.
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Advancing Disease Modeling in Animal-Based Research in Support of Precision Medicine: Proceedings of a Workshop Get This Book
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Precision medicine is focused on the individual and will require the rapid and accurate identification and prioritization of causative factors of disease. To move forward and accelerate the delivery of the anticipated benefits of precision medicine, developing predictable, reproducible, and reliable animal models will be essential. In order to explore the topic of animal-based research and its relevance to precision medicine, the National Academies of Sciences, Engineering, and Medicine convened a 2-day workshop on October 5 and 6, 2017. The workshop was designed to focus on the development, implementation, and interpretation of model organisms to advance and accelerate the field of precision medicine. Participants examined the extent to which next-generation animal models, designed using patient data and phenotyping platforms targeted to reveal and inform disease mechanisms, will be essential to the successful implementation of precision medicine. This publication summarizes the presentations and discussions from the workshop.

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