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Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Miller, James R C; Pfister, Edith L; Liu, Wanzhao; Andre, Ralph; Träger, Ulrike; Kennington, Lori A; Lo, Kimberly; Dijkstra, Sipke; Macdonald, Douglas; Ostroff, Gary; Aronin, Neil; Tabrizi, Sarah J.
Afiliación
  • Miller JRC; Department of Neurodegenerative Disease, UCL Institute of Neurology, University College London, London, WC1N 3BG, UK.
  • Pfister EL; RNA Therapeutics Institute and Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
  • Liu W; RNA Therapeutics Institute and Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
  • Andre R; Department of Neurodegenerative Disease, UCL Institute of Neurology, University College London, London, WC1N 3BG, UK.
  • Träger U; Department of Neurodegenerative Disease, UCL Institute of Neurology, University College London, London, WC1N 3BG, UK.
  • Kennington LA; RNA Therapeutics Institute and Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
  • Lo K; Charles River, Leiden, The Netherlands.
  • Dijkstra S; Charles River, Leiden, The Netherlands.
  • Macdonald D; CHDI Management/CHDI Foundation, Los Angeles, CA, USA.
  • Ostroff G; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
  • Aronin N; RNA Therapeutics Institute and Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA.
  • Tabrizi SJ; Department of Neurodegenerative Disease, UCL Institute of Neurology, University College London, London, WC1N 3BG, UK.
Sci Rep ; 7: 46740, 2017 04 24.
Article en En | MEDLINE | ID: mdl-28436437
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Enfermedad de Huntington / Polimorfismo de Nucleótido Simple / Proteínas Mutantes / Proteína Huntingtina Tipo de estudio: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Límite: Adult / Aged / Humans / Middle aged Idioma: En Revista: Sci Rep Año: 2017 Tipo del documento: Article País de afiliación: Reino Unido
Texto completo
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XML
PubMed Links
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Enfermedad de Huntington / Polimorfismo de Nucleótido Simple / Proteínas Mutantes / Proteína Huntingtina Tipo de estudio: Clinical_trials / Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Límite: Adult / Aged / Humans / Middle aged Idioma: En Revista: Sci Rep Año: 2017 Tipo del documento: Article País de afiliación: Reino Unido