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Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy.
Farrokhi, Vahid; Walsh, Jason; Palandra, Joe; Brodfuehrer, Joanne; Caiazzo, Teresa; Owens, Jane; Binks, Michael; Neelakantan, Srividya; Yong, Florence; Dua, Pinky; Le Guiner, Caroline; Neubert, Hendrik.
Afiliación
  • Farrokhi V; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 1 Burtt Road, Andover, MA, 01810, USA.
  • Walsh J; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 1 Burtt Road, Andover, MA, 01810, USA.
  • Palandra J; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 1 Burtt Road, Andover, MA, 01810, USA.
  • Brodfuehrer J; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 610 Main Street, Cambridge, MA, 02139, USA.
  • Caiazzo T; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 1 Burtt Road, Andover, MA, 01810, USA.
  • Owens J; Rare Disease Research Unit, Pfizer Worldwide Research & Development, 610 Main Street, Cambridge, MA, 02139, USA.
  • Binks M; Rare Disease Research Unit, Pfizer Worldwide Research & Development, 610 Main Street, Cambridge, MA, 02139, USA.
  • Neelakantan S; Clinical Pharmacology, Early Clinical Development, Worldwide Research & Development, Pfizer Inc, 1 Portland St, Cambridge, MA, 02139, USA.
  • Yong F; Biostatistics, Worldwide Research & Development, Pfizer Inc, Cambridge, MA, 02139, USA.
  • Dua P; Early Clinical Development, Clinical Pharmacology, Pfizer R&D UK Limited, Cambridge, UK.
  • Le Guiner C; Translational Gene Therapy Laboratory, University of Nantes, INSERM UMR1089, CHU de Nantes, IRS 2 Nantes Biotech, 22 Boulevard Benoni Goulin, 44200, Nantes, France.
  • Neubert H; Biomedicine Design, Worldwide Research & Development, Pfizer Inc, 1 Burtt Road, Andover, MA, 01810, USA. Hendrik.Neubert@pfizer.com.
Gene Ther ; 29(10-11): 608-615, 2022 11.
Article en En | MEDLINE | ID: mdl-34737451
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofina / Distrofia Muscular de Duchenne Límite: Animals / Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Distrofina / Distrofia Muscular de Duchenne Límite: Animals / Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido