Time: November 17, 2020 to January 19, 2021

Objectives: The workshop is organized by the Office of Research Infrastructure Program (ORIP) in collaboration with the National Heart, Lung, and Blood Institute (NHLBI), the National Institute on Aging (NIA), the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the National Institute of General Medical Sciences (NIGMS), and the National Institute of Neurological Disorders and Stroke (NINDS), and will be held as a series of sessions. This workshop will provide a venue to discuss the status and needs regarding the validation of animal models used in biomedical research. The focus of this workshop series will be on identifying obstacles, technology and resource gaps, and new approaches for assessing the value and limitations of animal models used to address basic science questions and/or to study human diseases.

Agenda

Session I. Validation of Invertebrate Models for Preclinical Research Time/Date: 2-4 p.m. ET on November 17, 2020 Chairs: Hugo Bellen, Baylor College of Medicine Julie Simpson, University of California Santa Barbara
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Julie Simpson, University of California Santa Barbara Goals of the invertebrate workshop Erik Jorgensen, University of Utah High-throughput gene tagging in C. elegans Gerald Rubin, Howard Hughes Medical Institute Stock Centers and high-resolution light and EM imaging tools Benjamin White, National Institute of Mental Health The potential benefits of GPCR biosensors, scarless split inteins, and methods for manipulating dense-core vesicle release Meng Wang, Baylor College of Medicine Metabolic imaging and profiling in model organisms Norbert Perrimon, Harvard Medical School Protein tagging, Nanobodies, proximity labeling methods and user friendly bioinformatic tools for data mining Greg Jefferis, MRC Laboratory of Molecular Biology An integrated molecular and connectomics atlas of brain cell types in Drosophila
3:30-4:00 p.m.	Group Discussion
Session II. Validation of Zebrafish Models for Preclinical Research Time/Date: 2-4 p.m. ET on November 24, 2020 Chairs: William Talbot, Stanford University Rebecca Burdine, Princeton University
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: William Talbot, Stanford University Zebrafish models of neurological disease Gerald Downes, University of Massachusetts Amherst Zebrafish Models of Genetic Epilepsies: Challenges and Opportunities Monte Westerfield, University of Oregon Zebrafish models validate undiagnosed human diseases Lilianna Solnica-Krezel, Washington University Forward and reverse genetic approaches in zebrafish to understanding scoliosis Leonard Zon, Boston Children's Hospital Cellular Barcoding in Zebrafish Models of Disease Lalita Ramakrishnan, University of Cambridge Zebrafish as models for infectious diseases Rebecca Burdine, Princeton University A New Era for Rare Disorders
3:30-4:00 p.m.	Group Discussion
Session III. Validation of Mouse Models for Preclinical Research Time/Date: 2-4 p.m. ET on December 1, 2020 Chairs: Cathleen Lutz, The Jackson Laboratory Douglas Wallace, Children's Hospital of Philadelphia
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Cathleen Lutz, The Jackson Laboratory Validation of Mouse Models for Preclinical Research Catherine Kaczorowski, The Jackson Laboratory Reproducible animal models for complex human disease: implications for personalized medicine Leonard Shultz, The Jackson Laboratory Next Generation Humanized Mice in Biomedical Research Kenneth Chien, Karolinska Institutet, Sweden Humanized mouse models for cardiovascular regenerative therapeutics Leonard Guarente, MIT Development of a Drug Discovery Platform for Dysferlinopathy-Based Muscular Dystrophies
3:30-4:00 p.m.	Group Discussion
Session IV. Validation of Large Animal Models for Preclinical Research Time/Date: 2-4 p.m. ET on December 8, 2020 Chairs: Jill Weimer, Sanford Research Susan Sanchez, University of Georgia
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Jill Weimer, Sanford Research Introduction to the Validation of Large Animal Models in Research Randall Prather, University of Missouri Swine as models of human disease and a source of organs for xenotransplantation Franklin West, University of Georgia The Pig Stroke Model: Evaluating neuroprotective and regenerative therapies Jessica Sieren, University of Iowa The Mutualistic Relationship between Medical Imaging and Large Animal Models Peter Nghiem, Texas A&M University Therapeutic Development in the Canine Models for Duchenne Muscular Dystrophy Duncan Lascelles, North Carolina State University Companion Animal Models of Chronic Pain Susan Sanchez, University of Georgia Validation of Large Animal Models in Research, a Summary
3:30-4:00 p.m.	Group Discussion
Session V. Validation of Non-Mouse Rodent Models for Preclinical Research Time/Date: 2-4 p.m. ET on December 10, 2020 Chairs: Mary Dickinson, Baylor College of Medicine Kent Lloyd, University of California Davis
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Renzhi Han, The Ohio State University A rabbit model of Duchenne muscular dystrophy for preclinical therapeutic testing Rochelle Buffenstein, Calico Life Sciences Breaking the mol(e)d; ther naked mole-rat a. long-lived animal model of exceptional biogerontological interest Hailey Weerts, Walter Reed Army Institute of Research Use of guinea pig models to assess the efficacy of enteric countermeasures John Engelhardt, University of Iowa Genetic Modeling in the Ferret to Study Disease Pathophysiology, Stem Cell Biology, and Genetic Therapies Rebecca Shansky, Northeastern University The Promise and Pitfalls of Rodent Models of Psychiatric Disease Johan Neyts, Katholieke Universiteit Leuven-Belgium Use of guinea pig models to assess the efficacy of enteric countermeasures
3:30-4:00 p.m.	Group Discussion
Session VI. Validation of Nonhuman Primate Models for Preclinical Research Time/Date: 2-4 p.m. ET on December 15, 2020 Chairs: John Morrison, University of California Davis David O’Connor, University of Wisconsin-Madison
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Jeff Rogers, Baylor College of Medicine Naturally occurring primate models of human genetic diseases: An under-exploited resource Amy Hartman, University of Pittsburg Advances in physiological monitoring of emerging viral diseases in non-human primates Erika Sasaki, Keio University What do we need for the validation of disease model marmosets? Diogo Magnani, University of Massachusetts Development of Species-Specific Therapies and Reagents at the Nonhuman Primate Reagent Resource (NHPRR) Jeff Kordower, Rush University Nonhuman primate models of Neurodegenerative diseases: Parkinson’s disease, Multiple System Atrophy, and Alzheimer’s disease Deborah L Fuller, University of Washington Challenges and advances in nonhuman primate models of respiratory infections
3:30-4:00 p.m.	Group Discussion
Session VII. Validation of Non-Zebrafish Aquatic Models for Preclinical Research Time/Date: 2-4 p.m. ET on Dec. 17, 2020 Chairs: John Postlethwait, University of Oregon Crystal Rogers, University of California, Davis
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: John Postlethwait, University of Oregon Introduction to the Validation of Aquatic Models in Research Manfred Schartl, Texas State University, San Marcos Xiphophorus, an evolutionary model for epistatic interactions in disease Kathryn Milligan-Myhre, University of Connecticut Adapting the evolutionary model organism, threespine stickleback, for host-microbe interaction studies Nicolas Rohner, University of Kansas Cavefish as a model for natural resilience to metabolic disease Matthew Harris, Boston Children's Hospital Strength in diversity: deep phylogenetic mapping to understand the causes and compensation of disease-related phenotypes Carole LaBonne, Northwestern University Xenopus, a powerful vertebrate model for biomedical research and modeling human disease Elly Tanaka, Research Institute of Molecular Pathology, Austria Genomics and phenomics of complex tissue regeneration using salamander model systems Lynne Fieber, University of Miami More alike than not: exploring neural models of human aging and neurodegenerative diseases in Aplysia
3:30-4:00 p.m.	Group Discussion
Session VIII. Technologies, Phenotyping, and Data Science for Animal Models Time/Date: 2-4 p.m. ET on January 5, 2021 Chairs: Keith Cheng, Penn State College of Medicine Stephen Ekker, Mayo Clinic
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Keith Cheng, Penn State College of Medicine Pan-cellular, Computational, and Integrative Tissue Phenomics as a Framework for Cross-species, Multi-scale Multi-omics Scott Fraser, University of Southern California Multiplex and Intravital Imaging for Model Validation Bo Zhang, Peking University Real-Time Genotyping and Phenotyping in Live Embryos for Disease Modeling and Validation Bo Zhang, Peking University Molecular Phenotyping of Live Embryos: Current Approaches and Challenges Lisa Schimmenti, Mayo Clinic Clinician and Patient Roles in Validating Animal Models of Human Disease Vasant Honavar, Pennsylvania State University The Promise and Potential of AI, Machine Learning and Data Science in Integrating Data and Models Across Modalities, Scales, and Model Systems Stephen Ekker, Mayo Clinic Illuminating the Genome Using Protein Trap Mutagenesis
3:30-4:00 p.m.	Group Discussion
Session IX. Vertical Integration Approach for Preclinical Research Time/Date: 2-4 p.m. ET on January 12, 2021 Chairs: Hugo Bellen, Baylor College of Medicine Calum A. MacRae, Brigham and Women's Hospital
2:00-2:05 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH
2:05-3:30 p.m.	Presentations: Hugo Bellen and Zhandong Liu, Baylor College of Medicine Modeling human variants in Drosophila and other model organisms and the integration of data from numerous sources for variant prioritization John Hogenesch, Cincinnati Children’s Hospital Medical Center Data Integration and validation of candidate variants regulating sleep and rhythms in cells and mice Olga Troyanskaya, Princeton University From genomes to networks - integrative cross-organism approaches to the study of human disease Peter Robinson, The Jackson Laboratory Knowledge graphs for data integration and machine learning in cross species disease research Rada Savic, University of California San Francisco Translational Tools and Cross-Species Data Integration Approaches for multi-drug regimen development for infectious diseases
3:30-4:00 p.m.	Group Discussion
Summary Session. Report and Discussion of Sessions I to IX Time/Date: 1-4 p.m. ET on Jan. 19, 2021 Chairs: Hugo Bellen, Baylor College of Medicine Keith Cheng, Penn State College of Medicine
1:00-1:10 p.m.	Opening Remarks Sige Zou, Program Official, ORIP, NIH Franziska Grieder, Director, ORIP, NIH Stephanie Murphy, Division Director, ORIP, NIH Hugo Bellen, Baylor College of Medicine Keith Cheng, Penn State College of Medicine
1:10-2:40 p.m.	Presentations: Hugo Bellen, Baylor College of Medicine, and Julie Simpson, University of California Santa Barbara Summary of Session I on Validation of Invertebrate Models for Preclinical Research William Talbot, Stanford University, and Rebecca Burdine, Princeton University Summary of Session II on Validation of Zebrafish Models for Preclinical Research Cathleen Lutz, The Jackson Laboratory, and Douglas Wallace, Children's Hospital of Philadelphia Summary of Session III on Validation of Mouse Models for Preclinical Research Jill Weimer, Sanford Research, and Susan Sanchez, University of Georgia Summary of Session IV on Validation of Large Animal Models for Preclinical Research Mary Dickinson, Baylor College of Medicine Kent Lloyd, University of California Davis Summary of Session V on Validation of Non-Mouse Rodent Models for Preclinical Research John Morrison, University of California Davis, and David O’Connor, University of Wisconsin-Madison Summary of Session VI on Validation of Nonhuman Primate Models for Preclinical Research John Postlethwait, University of Oregon, and Crystal Rogers, University of California, Davis Summary of Session VII on Validation of Non-Zebrafish Aquatic Models for Preclinical Research Keith Cheng, Penn State College of Medicine, and Stephen Ekker, Mayo Clinic Summary of Session VIII on Technologies, Phenotyping, and Data Science for Animal Models Hugo Bellen, Baylor College of Medicine, and Calum A. MacRae, Brigham and Women's Hospital Summary of Session IX on Vertical Integration Approach for Preclinical Research
2:40-4:00 p.m.	Group Discussion

Organization Committee
Subject Matter Experts
Hugo Bellen, Chair Keith Cheng, Co-Chair Alan Attie Stefania Forner Kent Lloyd Cathleen Lutz John Morrison Stacey Rizzo William Talbot Paul Territo Douglas Wallace Jill Weimer	Baylor College of Medicine Penn State College of Medicine University of Wisconsin -Madison University of California, Irvine University of California, Davis Jackson Laboratory University of California, Davis University of Pittsburgh Stanford University Indiana University Children’s Hospital of Philadelphia Sanford Research
NIH Program Staff
Sige Zou, Coordinator Kristine Abraham Shreaya Chakroborty Marc Charette Miguel Contreras Bruce Fuchs Amelie Gubitz Lisa Schwartz Longacre D.P. Mohapatra Lorenzo M. Refolo Rebecca Roof Xiaoli Zhao	ORIP/DPCPSI/OD/NIH NIDDK/NIH NIA/NIH NHLBI/NIH ORIP/DPCPSI/OD/NIH ORIP/DPCPSI/OD/NIH NINDS/NIH NHLBI/NIH NINDS/NIH NIA/NIH NINDS/NIH NIGMS/NIH
NIH Supporting Staff
Lola Ajayi Desiree von Kollmar Susan Chandran	ORIP/DPCPSI/OD/NIH ORIP/DPCPSI/OD/NIH ORIP/DPCPSI/OD/NIH