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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Davidson, Beverly L; Gao, Guangping; Berry-Kravis, Elizabeth; Bradbury, Allison M; Bönnemann, Carsten; Buxbaum, Joseph D; Corcoran, Gavin R; Gray, Steven J; Gray-Edwards, Heather; Kleiman, Robin J; Shaywitz, Adam J; Wang, Dan; Zoghbi, Huda Y; Flotte, Terence R; Tauscher-Wisniewski, Sitra; Tifft, Cynthia J; Sahin, Mustafa.
Affiliation
  • Davidson BL; 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: davidsonbl@chop.edu.
  • Gao G; 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: guangping.g
  • Berry-Kravis E; Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL, USA.
  • Bradbury AM; Department of Pediatrics, Abigail Wexner Research Institute Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
  • Bönnemann C; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
  • Buxbaum JD; Seaver Autism Center for Research and Treatment, Department of Psychiatry, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Corcoran GR; TrialSpark, New York, NY, USA.
  • Gray SJ; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
  • Gray-Edwards H; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
  • Kleiman RJ; Biogen Research and Development, Cambridge, MA, USA.
  • Shaywitz AJ; BridgeBio Gene Therapy, Palo Alto, CA, USA.
  • Wang D; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, USA.
  • Zoghbi HY; Howard Hughes Medical Institute, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
  • Flotte TR; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA.
  • Tauscher-Wisniewski S; Novartis Gene Therapies, Bannockburn, IL, USA.
  • Tifft CJ; Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Sahin M; Rosamund Stone Zander Translational Neuroscience Center, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA. Electronic address: mustafa.sahin@childrens.harvard.edu.
Mol Ther ; 30(7): 2416-2428, 2022 07 06.
Article in En | MEDLINE | ID: mdl-35585789
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Precision Medicine / Mental Disorders Type of study: Diagnostic_studies Limits: Humans Country/Region as subject: America do norte Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2022 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Precision Medicine / Mental Disorders Type of study: Diagnostic_studies Limits: Humans Country/Region as subject: America do norte Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2022 Document type: Article