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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
Affiliation:1. Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children’s Hospital, Columbus, OH 43205, USA;2. Neuroscience Graduate Program, The Ohio State University, Columbus, OH 43210, USA;3. Simons Initiative for the Developing Brain, Centre for Discovery Brain Sciences, University of Edinburgh, EH8 9XD Edinburgh, UK;4. The Mannheimer Foundation Inc, Homestead, FL 33034, USA;5. The Department of Pathology & Laboratory Medicine, Nationwide Children’s Hospital, Columbus, OH 43205, USA;6. Department of Pathology and the Department of Biomedical Education & Anatomy, The Ohio State University, Columbus, OH 43210, USA;7. GEMpath, Inc, Longmont, CO 80504, USA;8. Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA
Abstract:
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  • Keywords:Rett syndrome  gene therapy  adeno-associated virus serotype 9, scAAV9  methyl-CpG binding protein 2, MECP2  P546
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