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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers, Samantha; Likhite, Shibi; Gadalla, Kamal K; Miranda, Carlos J; Huffenberger, Amy J; Dennys, Cassandra; Foust, Kevin D; Morales, Pablo; Pierson, Christopher R; Rinaldi, Federica; Perry, Stephanie; Bolon, Brad; Wein, Nicolas; Cobb, Stuart; Kaspar, Brian K; Meyer, Kathrin C.
Afiliación
  • Powers S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA; Neuroscience Graduate Program, The Ohio State University, Columbus, OH 43210, USA. Electronic address: Samantha.powers@nationwidechildrens.org.
  • Likhite S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Gadalla KK; Simons Initiative for the Developing Brain, Centre for Discovery Brain Sciences, University of Edinburgh, EH8 9XD Edinburgh, UK.
  • Miranda CJ; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Huffenberger AJ; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Dennys C; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Foust KD; Neuroscience Graduate Program, The Ohio State University, Columbus, OH 43210, USA.
  • Morales P; The Mannheimer Foundation Inc, Homestead, FL 33034, USA.
  • Pierson CR; The Department of Pathology & Laboratory Medicine, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pathology and the Department of Biomedical Education & Anatomy, The Ohio State University, Columbus, OH 43210, USA.
  • Rinaldi F; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Perry S; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Bolon B; GEMpath, Inc, Longmont, CO 80504, USA.
  • Wein N; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.
  • Cobb S; Simons Initiative for the Developing Brain, Centre for Discovery Brain Sciences, University of Edinburgh, EH8 9XD Edinburgh, UK.
  • Kaspar BK; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.
  • Meyer KC; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH 43205, USA; Neuroscience Graduate Program, The Ohio State University, Columbus, OH 43210, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA. Electronic address: Ka
Mol Ther ; 31(9): 2767-2782, 2023 09 06.
Article en En | MEDLINE | ID: mdl-37481701
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Síndrome de Rett Tipo de estudio: Clinical_trials Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Síndrome de Rett Tipo de estudio: Clinical_trials Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos