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Allele-specific silencing as treatment for gene duplication disorders: proof-of-principle in autosomal dominant leukodystrophy.
Giorgio, Elisa; Lorenzati, Martina; Rivetti di Val Cervo, Pia; Brussino, Alessandro; Cernigoj, Manuel; Della Sala, Edoardo; Bartoletti Stella, Anna; Ferrero, Marta; Caiazzo, Massimiliano; Capellari, Sabina; Cortelli, Pietro; Conti, Luciano; Cattaneo, Elena; Buffo, Annalisa; Brusco, Alfredo.
Afiliación
  • Giorgio E; University of Torino, Department of Medical Sciences, Torino, Italy.
  • Lorenzati M; University of Torino, Department of Neuroscience Rita Levi Montalcini and Neuroscience Institute Cavalieri Ottolenghi (NICO), Orbassano, Torino, Italy.
  • Rivetti di Val Cervo P; University of Milan, Department of Biosciences, Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases, Milan, Italy.
  • Brussino A; University of Torino, Department of Medical Sciences, Torino, Italy.
  • Cernigoj M; University of Milan, Department of Biosciences, Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases, Milan, Italy.
  • Della Sala E; University of Torino, Department of Medical Sciences, Torino, Italy.
  • Bartoletti Stella A; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bellaria Hospital, Bologna, Italy.
  • Ferrero M; University of Torino, Department of Medical Sciences, Torino, Italy.
  • Caiazzo M; Department of Pharmaceutics, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Universiteitsweg 99, CG, Utrecht, The Netherlands.
  • Capellari S; Department of Molecular Medicine and Medical Biotechnology, University of Naples 'Federico II', Naples, Italy.
  • Cortelli P; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bellaria Hospital, Bologna, Italy.
  • Conti L; University of Bologna, Department of Biomedical and Neuromotor Sciences, Bologna, Italy.
  • Cattaneo E; IRCCS Istituto delle Scienze Neurologiche di Bologna, Bellaria Hospital, Bologna, Italy.
  • Buffo A; University of Bologna, Department of Biomedical and Neuromotor Sciences, Bologna, Italy.
  • Brusco A; University of Trento, Centre for Integrative Biology (CIBIO), Laboratory of Computational Oncology, Trento, Italy.
Brain ; 142(7): 1905-1920, 2019 07 01.
Article en En | MEDLINE | ID: mdl-31143934
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Duplicación de Gen / Enfermedad de Pelizaeus-Merzbacher / Silenciador del Gen / Lamina Tipo B / ARN Interferente Pequeño / Alelos / Enfermedades Genéticas Congénitas Tipo de estudio: Observational_studies / Risk_factors_studies Límite: Animals / Humans Idioma: En Año: 2019 Tipo del documento: Article
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Duplicación de Gen / Enfermedad de Pelizaeus-Merzbacher / Silenciador del Gen / Lamina Tipo B / ARN Interferente Pequeño / Alelos / Enfermedades Genéticas Congénitas Tipo de estudio: Observational_studies / Risk_factors_studies Límite: Animals / Humans Idioma: En Año: 2019 Tipo del documento: Article