Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

Affiliations

• ¹ Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: [email protected].
• ² Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, UMass Chan Medical School, Worcester, MA, USA. Electronic address: [email protected].
• ³ Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL, USA.
• ⁴ Department of Pediatrics, Abigail Wexner Research Institute Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
• ⁵ National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
• ⁶ Seaver Autism Center for Research and Treatment, Department of Psychiatry, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
• ⁷ TrialSpark, New York, NY, USA.
• ⁸ Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
• ⁹ Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
• ¹⁰ Biogen Research and Development, Cambridge, MA, USA.
• ¹¹ BridgeBio Gene Therapy, Palo Alto, CA, USA.
• ¹² Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, USA.
• ¹³ Howard Hughes Medical Institute, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
• ¹⁴ Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA.
• ¹⁵ Novartis Gene Therapies, Bannockburn, IL, USA.
• ¹⁶ Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
• ¹⁷ Rosamund Stone Zander Translational Neuroscience Center, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA. Electronic address: [email protected].