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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.
Afiliación
  • 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 en En | MEDLINE | ID: mdl-35585789
ABSTRACT
We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Medicina de Precisión / Trastornos Mentales Tipo de estudio: Diagnostic_studies Límite: Humans País/Región como asunto: America do norte Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2022 Tipo del documento: Article
Texto completo
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Medicina de Precisión / Trastornos Mentales Tipo de estudio: Diagnostic_studies Límite: Humans País/Región como asunto: America do norte Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2022 Tipo del documento: Article