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1 Introduction and Background
Pages 1-6

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From page 1...
... While research with rodent models has led to significant medical discoveries and increased fundamental understanding of brain function, pathology, and disease pathogenesis through basic science research, there are limitations to their use in studying human nervous system disorders, including the vast differences in brain structure and their inability to model many aspects of normal human cognition and behavior (IOM, 2013)
From page 2...
... , and also to introduce scientific and bioethical questions that merit deep consideration.2 To examine the promise, concerns, and challenges related to neuroscience research using genetically modified nonhuman primates, the Forum on Neuroscience and Nervous System Disorders hosted a public workshop on October 4, 2018, bringing together an international 2In September 2016, the National Institutes of Health hosted a congressionally requested workshop on Ensuring the Continued Responsible Oversight of Research with Non-Human Primates to review the appropriateness of research in nonhuman primates, including rationale and guidelines. For more information, go to (accessed December 13, 2018)
From page 3...
... Yet, the field must still grapple with the question of what merits taking the step of developing nonhuman primate models with genetic modifications, and if these models are deemed to be appropriate and essential, how the field will ensure the appropriate use of this extremely unique resource, said Jensen. For a comprehensive look at the issues, the workshop considered some of the distinct aspects of animal husbandry required to ensure optimal care of genetically modified nonhuman primates; the ethical considerations related to the use of these animals in research and the global regulatory and conduct codes that are in place or that need to be developed; and the infrastructure required worldwide to ensure that research achieves its greatest scientific impact through collaboration and partnerships.
From page 4...
... and explore how they would need to be mod ified for use in other animal models, such as nonhuman primates. • Consider bioethical principles and issues related to genetic engi neering of animal models for nervous system disorders, and dis cuss potential metrics for determining the models' readiness for nonhuman primate research.
From page 5...
... ORGANIZATION OF THE PROCEEDINGS Chapter 2 explores the rationale for developing genetically modified nonhuman primate models of nervous system disorders and provides an overview of challenges and opportunities associated with this area of research for academia, industry, and the public. Chapter 3 describes the development of several nonhuman primate models of human diseases using genetic modification and highlights a case study of how viral delivery of genes was used to develop the first successful gene therapy of a central nervous system disease.

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