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Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action.
Laselva, Onofrio; Eckford, Paul Dw; Bartlett, Claire; Ouyang, Hong; Gunawardena, Tarini Na; Gonska, Tanja; Moraes, Theo J; Bear, Christine E.
  • Laselva O; Programme in Molecular Medicine, Hospital for Sick Children Research Institute, Toronto, Canada; Department of Physiology, University of Toronto, Toronto, Canada.
  • Eckford PD; Programme in Molecular Medicine, Hospital for Sick Children Research Institute, Toronto, Canada.
  • Bartlett C; Programme in Translational Medicine, Hospital for Sick Children Research Institute, Toronto, Canada.
  • Ouyang H; Programme in Translational Medicine, Hospital for Sick Children Research Institute, Toronto, Canada.
  • Gunawardena TN; Programme in Translational Medicine, Hospital for Sick Children Research Institute, Toronto, Canada.
  • Gonska T; Programme in Translational Medicine, Hospital for Sick Children Research Institute, Toronto, Canada; Department of Paediatrics, University of Toronto, Toronto, Canada.
  • Moraes TJ; Programme in Translational Medicine, Hospital for Sick Children Research Institute, Toronto, Canada; Department of Paediatrics, University of Toronto, Toronto, Canada. Electronic address: theo.moraes@sickkids.ca.
  • Bear CE; Programme in Molecular Medicine, Hospital for Sick Children Research Institute, Toronto, Canada; Department of Physiology, University of Toronto, Toronto, Canada; Department of Biochemistry, University of Toronto, Toronto, Canada. Electronic address: bear@sickkids.ca.
J Cyst Fibros ; 19(5): 717-727, 2020 09.
Article en En | MEDLINE | ID: mdl-31831337
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Codón sin Sentido / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Células Epiteliales / Degradación de ARNm Mediada por Codón sin Sentido Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Codón sin Sentido / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Células Epiteliales / Degradación de ARNm Mediada por Codón sin Sentido Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article