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In search of a cure: PACS1 Research Foundation as a model of rare disease therapy development
Affiliation:1. Department of Neuroscience, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA;2. Northwestern University Interdepartmental Neuroscience (NUIN) Graduate Program, Northwestern University, Chicago, IL, USA;3. PACS1 Syndrome Research Foundation, Old Greenwich, CT, USA;1. Neurometabolic Unit and Synaptic Metabolism Laboratory, Department of Neurology, IPR, CIBERER and MetabERN, Hospital Sant Joan de Déu, Barcelona, Spain;2. Groupe de Recherche Clinique Neurométabolique, Université Pierre et Marie Curie, 22 rue Juliette Lamber Paris, 75017, France;3. Division of Metabolic Diseases, Bambino Gesù Children’s Hospital IRCCS, Rome, Italy;1. Vanderbilt Genetics Institute, Vanderbilt University, Nashville, TN 37235, USA;2. Department of Molecular Physiology and Biophysics, Vanderbilt University, Nashville, TN 37235, USA;3. Comprehensive Cancer Center, University of New Mexico, Albuquerque, NM 87131, USA;1. Gene Editing Center, School of Life Science and Technology, ShanghaiTech University, Shanghai 201210, China;2. Zhejiang Provincial Key Laboratory of Pancreatic Disease, The First Affiliated Hospital, and Institute of Translational Medicine, Zhejiang University School of Medicine, Hangzhou 310029, China;1. Integrative Genomics of Ageing Group, Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, UK;1. Molecular Virology Laboratory, School of Life Sciences, Jawaharlal Nehru University, New Delhi 110067, India;1. ARC Centre of Excellence in Synthetic Biology, Macquarie University, Sydney, NSW 2109, Australia;2. Department of Molecular Sciences, Macquarie University, Sydney, NSW 2109, Australia;3. New South Wales Department of Primary Industries, Orange, NSW 2800, Australia
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