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Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A.
Long, Brian R; Veron, Philippe; Kuranda, Klaudia; Hardet, Romain; Mitchell, Nina; Hayes, Gregory M; Wong, Wing Yen; Lau, Kelly; Li, Mingjin; Hock, M Benjamin; Zoog, Stephen J; Vettermann, Christian; Mingozzi, Federico; Schweighardt, Becky.
Afiliação
  • Long BR; BioMarin Pharmaceutical Inc., Novato, CA, USA. Electronic address: brian.long@bmrn.com.
  • Veron P; University Pierre and Marie Curie and INSERM U974, Paris, France; Genethon, 91000, Evry, France; Université Paris-Saclay, Univ Evry, INSERM, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.
  • Kuranda K; University Pierre and Marie Curie and INSERM U974, Paris, France.
  • Hardet R; University Pierre and Marie Curie and INSERM U974, Paris, France.
  • Mitchell N; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Hayes GM; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Wong WY; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Lau K; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Li M; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Hock MB; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Zoog SJ; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Vettermann C; BioMarin Pharmaceutical Inc., Novato, CA, USA.
  • Mingozzi F; University Pierre and Marie Curie and INSERM U974, Paris, France; Genethon, 91000, Evry, France; Université Paris-Saclay, Univ Evry, INSERM, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France.
  • Schweighardt B; BioMarin Pharmaceutical Inc., Novato, CA, USA.
Mol Ther ; 29(2): 597-610, 2021 02 03.
Article em En | MEDLINE | ID: mdl-33309883

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos / Hemofilia A Limite: Adult / Humans / Male Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos / Hemofilia A Limite: Adult / Humans / Male Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article
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