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Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.
De, Bishnu P; Rosenberg, Jonathan B; Selvan, Nithya; Wilson, Isabelle; Yusufzai, Nadir; Greco, Alessandria; Kaminsky, Stephen M; Heier, Linda A; Ricart Arbona, Rodolfo J; Miranda, Ileana C; Monette, Sebastien; Nair, Anju; Khanna, Richie; Crystal, Ronald G; Sondhi, Dolan.
Afiliación
  • De BP; Department of Genetic Medicine, New York, New York, USA.
  • Rosenberg JB; Department of Genetic Medicine, New York, New York, USA.
  • Selvan N; LEXEO Therapeutics, New York, New York, USA.
  • Wilson I; Department of Genetic Medicine, New York, New York, USA.
  • Yusufzai N; Department of Genetic Medicine, New York, New York, USA.
  • Greco A; Department of Genetic Medicine, New York, New York, USA.
  • Kaminsky SM; Department of Genetic Medicine, New York, New York, USA.
  • Heier LA; Department of Radiology, Weill Cornell Medical College, 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.
  • Miranda IC; Laboratory of Comparative Pathology, Memorial Sloan Kettering Cancer Center, The Rockefeller University, Weill Cornell Medicine, New York, New York, USA.
  • Monette S; Laboratory of Comparative Pathology, Memorial Sloan Kettering Cancer Center, The Rockefeller University, Weill Cornell Medicine, New York, New York, USA.
  • Nair A; LEXEO Therapeutics, New York, New York, USA.
  • Khanna R; 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(17-18): 905-916, 2023 09.
Article en En | MEDLINE | ID: mdl-37624739
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Lipofuscinosis Ceroideas Neuronales Tipo de estudio: Clinical_trials Límite: Animals / Child / 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
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Lipofuscinosis Ceroideas Neuronales Tipo de estudio: Clinical_trials Límite: Animals / Child / 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