Transgenic and Chimeric Neuroscience Research: Exploring the Scientific Opportunities Afforded by New Nonhuman Primate Models — A Workshop

When: October 4, 2018 (8:30 AM Eastern)
Where: Keck Center of the National Academies of Sciences, Engineering, and Medicine (Room 100) • 500 Fifth Street NW, Washington, D.C. 20001

Topics Biomedical and Health Research, Diseases, Public Health, Quality and Patient Safety
Activity: Forum on Neuroscience and Nervous System Disorders
Board: Board on Health Sciences Policy




The translational disconnect from preclinical studies with predominantly rodent animal models to human clinical trials remains a key challenge associated with lagging development of therapies for brain disorders. Since 2012, the Forum has hosted a series of workshops examining different aspects of this challenge, including maximizing the translation of effective therapies from animal models to clinical practice and exploring the evidence needed to bring compounds that appear to be safe into human efficacy trials. While no animal model will fully recapitulate human nervous system disorders, nonhuman primates—such as marmosets and macaques—have shown promise in their ability to serve as models for complex brain disorders, given the phylogenetic proximity and genetic similarity to humans, similarity of neuroanatomical organization (e.g., a well-developed prefrontal cortex) and associated cognitive and behavioral functions, social cognition, and the ability to study developmental phenotypes and prodromal disease states. 


Workshop Objectives: 

This one-day public workshop will bring together experts and key stakeholders from academia, government, industry, and non-profit organizations to examine the scientific opportunities and challenges, as well as bioethical considerations, of genetically engineered nonhuman primate models for neuroscience research. Invited presentations and discussions will be designed to:

  • Discuss the state of the science of transgenic and chimeric neuroscience research and emerging models for nervous system disorders, and explore the potential usefulness of such models to enhance understanding of behavior and higher cortical function, and advance therapeutic development.
  • Examine current tools and technologies used in rodent models (e.g., transgenesis, chimera, AAVs [adeno-associated viruses], gene therapy, etc.) and explore how they would need to be modified for use in other animal models, such as nonhuman primates.
  • Consider bioethical principles and issues related to genetic engineering of animal models for nervous system disorders, and discuss potential metrics for determining the models’ readiness for nonhuman primate research.
  • Discuss policies and infrastructure needed to advance research in this domain including, for example, training, recruitment of early career scientists, and the potential development of specialized research centers and international collaborations.


Related Event: Care, Use and Welfare of Marmosets as Animal Models for Gene Editing-based Biomedical Research  October 22-23, 2018 in Washington, DC

The Roundtable on Science and Welfare in Laboratory Animal Use will hold a workshop to explore and address concerns related to the increasing popularity of marmosets as animal models. The workshop will feature discussions on the availability and genetic diversity of marmosets; presentations on their use as research models; elements of marmoset husbandry and veterinary care; and ethical and welfare considerations regarding the use of marmosets in research. Invited speakers and discussants will contribute perspectives from government, academia, industry, and nonprofit sectors at the global and national level. For more information, please click here.  

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