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Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
Munoz-Zuluaga, Carlos; Gertz, Monica; Yost-Bido, Melissa; Greco, Alessandria; Gorman, Nicholas; Chen, Alvin; Kooner, Vikrum; Rosenberg, Jonathan B; De, Bishnu P; Kaminsky, Stephen M; Borczuk, Alain; Ricart Arbona, Rodolfo J; Martin, Heather R; Monette, Sebastien; Khanna, Richie; Barth, Jay A; Crystal, Ronald G; Sondhi, Dolan.
Afiliación
  • Munoz-Zuluaga C; Department of Genetic Medicine, New York, New York, USA.
  • Gertz M; Department of Genetic Medicine, New York, New York, USA.
  • Yost-Bido M; Department of Genetic Medicine, New York, New York, USA.
  • Greco A; Department of Genetic Medicine, New York, New York, USA.
  • Gorman N; Department of Genetic Medicine, New York, New York, USA.
  • Chen A; Department of Genetic Medicine, New York, New York, USA.
  • Kooner V; Department of Genetic Medicine, New York, New York, USA.
  • Rosenberg JB; Department of Genetic Medicine, New York, New York, USA.
  • De BP; Department of Genetic Medicine, New York, New York, USA.
  • Kaminsky SM; Department of Genetic Medicine, New York, New York, USA.
  • Borczuk A; Department of Pathology, Weill Cornell Medicine, New York, New York, USA.
  • Ricart Arbona RJ; Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, New York, New York, USA.
  • Martin HR; Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, New York, New York, USA.
  • Monette S; Center for Comparative Medicine and Pathology, Memorial Sloan Kettering Cancer Center, Weill Cornell Medicine, New York, New York, USA.
  • Khanna R; LEXEO Therapeutics, New York, New York, USA.
  • Barth JA; LEXEO Therapeutics, New York, New York, USA.
  • Crystal RG; Department of Genetic Medicine, New York, New York, USA.
  • Sondhi D; Department of Genetic Medicine, New York, New York, USA.
Hum Gene Ther ; 34(13-14): 605-615, 2023 07.
Article en En | MEDLINE | ID: mdl-37166361
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Ataxia de Friedreich / Insuficiencia Cardíaca Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Ataxia de Friedreich / Insuficiencia Cardíaca Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos