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Intronic CRISPR Repair in a Preclinical Model of Noonan Syndrome-Associated Cardiomyopathy.
Hanses, Ulrich; Kleinsorge, Mandy; Roos, Lennart; Yigit, Gökhan; Li, Yun; Barbarics, Boris; El-Battrawy, Ibrahim; Lan, Huan; Tiburcy, Malte; Hindmarsh, Robin; Lenz, Christof; Salinas, Gabriela; Diecke, Sebastian; Müller, Christian; Adham, Ibrahim; Altmüller, Janine; Nürnberg, Peter; Paul, Thomas; Zimmermann, Wolfram-Hubertus; Hasenfuss, Gerd; Wollnik, Bernd; Cyganek, Lukas.
Afiliación
  • Hanses U; Clinic for Cardiology and Pneumology (U.H., M.K., L.R., R.H., G.H., L.C.).
  • Kleinsorge M; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Roos L; Clinic for Cardiology and Pneumology (U.H., M.K., L.R., R.H., G.H., L.C.).
  • Yigit G; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Li Y; Clinic for Cardiology and Pneumology (U.H., M.K., L.R., R.H., G.H., L.C.).
  • Barbarics B; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • El-Battrawy I; Institute of Human Genetics (G.Y., Y.L., G.S., C.M., I.A., B.W.).
  • Lan H; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Tiburcy M; Institute of Human Genetics (G.Y., Y.L., G.S., C.M., I.A., B.W.).
  • Hindmarsh R; Clinic for Pediatric Cardiology and Intensive Care Medicine (B.B., T.P.).
  • Lenz C; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Salinas G; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Diecke S; First Department of Medicine, Medical Faculty Mannheim, University Medical Centre Mannheim, University of Heidelberg, Mannheim, Germany (I.E-B., H.L.).
  • Müller C; First Department of Medicine, Medical Faculty Mannheim, University Medical Centre Mannheim, University of Heidelberg, Mannheim, Germany (I.E-B., H.L.).
  • Adham I; Institute of Pharmacology and Toxicology (M.T., W-H.Z.).
  • Altmüller J; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Nürnberg P; Clinic for Cardiology and Pneumology (U.H., M.K., L.R., R.H., G.H., L.C.).
  • Paul T; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
  • Zimmermann WH; Institute for Clinical Chemistry (C.L.), University Medical Center Göttingen, Germany.
  • Hasenfuss G; Bioanalytical Mass Spectrometry, Max Planck Institute for Biophysical Chemistry, Göttingen, Germany (C.L.).
  • Wollnik B; Institute of Human Genetics (G.Y., Y.L., G.S., C.M., I.A., B.W.).
  • Cyganek L; DZHK (German Center for Cardiovascular Research), partner site Göttingen, Mannheim and Berlin, Germany (U.H., M.K., L.R., G.Y., B.B., I.E-B., M.T., R.H., S.D., T.P., W.-H.Z., G.H., B.W., L.C.).
Circulation ; 142(11): 1059-1076, 2020 09 15.
Article en En | MEDLINE | ID: mdl-32623905
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Factores de Transcripción / Terapia Genética / Miocitos Cardíacos / Células Madre Pluripotentes Inducidas / Sistemas CRISPR-Cas / Modelos Cardiovasculares / Mutación / Cardiomiopatías / Síndrome de Noonan Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article
Texto completo
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XML
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Factores de Transcripción / Terapia Genética / Miocitos Cardíacos / Células Madre Pluripotentes Inducidas / Sistemas CRISPR-Cas / Modelos Cardiovasculares / Mutación / Cardiomiopatías / Síndrome de Noonan Tipo de estudio: Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Año: 2020 Tipo del documento: Article