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In utero gene therapy rescues microcephaly caused by Pqbp1-hypofunction in neural stem progenitor cells.
Ito, H; Shiwaku, H; Yoshida, C; Homma, H; Luo, H; Chen, X; Fujita, K; Musante, L; Fischer, U; Frints, S G M; Romano, C; Ikeuchi, Y; Shimamura, T; Imoto, S; Miyano, S; Muramatsu, S-i; Kawauchi, T; Hoshino, M; Sudol, M; Arumughan, A; Wanker, E E; Rich, T; Schwartz, C; Matsuzaki, F; Bonni, A; Kalscheuer, V M; Okazawa, H.
Afiliação
  • Ito H; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Shiwaku H; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Yoshida C; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Homma H; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Luo H; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Chen X; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Fujita K; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
  • Musante L; Department of Human Molecular Genetics, Max-Planck Institute for Molecular Genetics, Berlin-Dahlem, Germany.
  • Fischer U; Department of Human Molecular Genetics, Max-Planck Institute for Molecular Genetics, Berlin-Dahlem, Germany.
  • Frints SG; 1] Department of Clinical Genetics, University Hospital azM Maastricht, Maastricht, The Netherlands [2] School for Oncology and Developmental Biology, GROW, Maastricht University, Maastricht, The Netherlands.
  • Romano C; Unità Operativa Complessa di Pediatria e Genetica Medica, IRCCS Associazione Oasi Maria Santissima, Troina (Enna), Italy.
  • Ikeuchi Y; 1] Department of Anatomy and Neurobiology, Washington University School of Medicine, St Louis, MO, USA [2] Department of Neurobiology, Harvard Medical School, Boston, MA, USA.
  • Shimamura T; Human Genome Center, Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
  • Imoto S; Human Genome Center, Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
  • Miyano S; Human Genome Center, Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
  • Muramatsu SI; Department of Neurology, Jichi Medical University, Tochigi, Japan.
  • Kawauchi T; Department of Anatomy, Keio University School of Medicine, Tokyo, Japan.
  • Hoshino M; Department of Biochemistry and Cellular Biology, National Center for Neurology and Psychiatry, Tokyo, Japan.
  • Sudol M; Laboratory of Signal Transduction and Proteomic Profiling, Weis Center for Research, Geisinger Clinic, Danville, PA, USA.
  • Arumughan A; Department of Neurogenetics, Max-Delbrück Center for Molecular Medicine, Berlin-Buch, Germany.
  • Wanker EE; Department of Neurogenetics, Max-Delbrück Center for Molecular Medicine, Berlin-Buch, Germany.
  • Rich T; Institute of Infection, Immunity and Inflammation, University of Glasgow, Glasgow, UK.
  • Schwartz C; JC Self Research Institute of Human Genetics, Greenwood Genetic Center, Greenwood, SC, USA.
  • Matsuzaki F; Laboratory for Cell Asymmetry, Center for Developmental Biology, RIKEN, Chuo-ku, Kobe, Japan.
  • Bonni A; 1] Department of Anatomy and Neurobiology, Washington University School of Medicine, St Louis, MO, USA [2] Department of Neurobiology, Harvard Medical School, Boston, MA, USA.
  • Kalscheuer VM; Department of Human Molecular Genetics, Max-Planck Institute for Molecular Genetics, Berlin-Dahlem, Germany.
  • Okazawa H; Department of Neuropathology, Medical Research Institute and Center for Brain Integration Research, Tokyo Medical and Dental University, Tokyo, Japan.
Mol Psychiatry ; 20(4): 459-71, 2015 Apr.
Article em En | MEDLINE | ID: mdl-25070536

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas Nucleares / Terapia Genética / Células-Tronco Neurais / Microcefalia Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2015 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas Nucleares / Terapia Genética / Células-Tronco Neurais / Microcefalia Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2015 Tipo de documento: Article