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Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Kleine Holthaus, Sophia-Martha; Herranz-Martin, Saul; Massaro, Giulia; Aristorena, Mikel; Hoke, Justin; Hughes, Michael P; Maswood, Ryea; Semenyuk, Olha; Basche, Mark; Shah, Amna Z; Klaska, Izabela P; Smith, Alexander J; Mole, Sara E; Rahim, Ahad A; Ali, Robin R.
Afiliação
  • Kleine Holthaus SM; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Herranz-Martin S; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK.
  • Massaro G; Centro de Biología Molecular Severo Ochoa (UAM-CSIC) and Departamento de Biología Molecular, Universidad Autónoma de Madrid (UAM), Madrid, Spain.
  • Aristorena M; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK.
  • Hoke J; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Hughes MP; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Maswood R; Department of Pharmacology, UCL School of Pharmacy, 29-39 Brunswick Square, Bloomsbury, London WC1N 1AX, UK.
  • Semenyuk O; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Basche M; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Shah AZ; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Klaska IP; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Smith AJ; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Mole SE; Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Rahim AA; MRC Laboratory for Molecular Cell Biology, University College London, Gower Street, London WC1E 6BT, UK.
  • Ali RR; UCL Institute of Child Health, 30 Guildford Street, London WC1N 1EH, UK.
Hum Mol Genet ; 28(23): 3867-3879, 2019 12 01.
Article em En | MEDLINE | ID: mdl-31807779
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Vetores Genéticos / Proteínas de Membrana / Lipofuscinoses Ceroides Neuronais Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Reino Unido País de publicação: Reino Unido
Texto completo
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XML
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Vetores Genéticos / Proteínas de Membrana / Lipofuscinoses Ceroides Neuronais Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Reino Unido País de publicação: Reino Unido