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Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
Le Guiner, Caroline; Xiao, Xiao; Larcher, Thibaut; Lafoux, Aude; Huchet, Corinne; Toumaniantz, Gilles; Adjali, Oumeya; Anegon, Ignacio; Remy, Séverine; Grieger, Josh; Li, Juan; Farrokhi, Vahid; Neubert, Hendrik; Owens, Jane; McIntyre, Maritza; Moullier, Philippe; Samulski, R Jude.
Afiliação
  • Le Guiner C; Nantes Université, CHU Nantes, INSERM, TaRGeT, UMR 1089, Translational Research for Gene Therapies, 44200 Nantes, France.
  • Xiao X; Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599-7352, USA.
  • Larcher T; INRAE Oniris, UMR 703, PanTher, APEX, 44307 Nantes, France.
  • Lafoux A; Therassay Platform, Capacités, Nantes Université, 44007 Nantes, France.
  • Huchet C; Nantes Université, CHU Nantes, INSERM, TaRGeT, UMR 1089, Translational Research for Gene Therapies, 44200 Nantes, France.
  • Toumaniantz G; Therassay Platform, Capacités, Nantes Université, 44007 Nantes, France.
  • Adjali O; Therassay Platform, Capacités, Nantes Université, 44007 Nantes, France.
  • Anegon I; Nantes Université, CHU Nantes, CNRS, L'Institut du Thorax, 44007 Nantes, France.
  • Remy S; Nantes Université, CHU Nantes, INSERM, TaRGeT, UMR 1089, Translational Research for Gene Therapies, 44200 Nantes, France.
  • Grieger J; Nantes Université, CHU Nantes, INSERM, Center for Research in Transplantation and Translational Immunology, UMR 1064, ITUN, 44093 Nantes, France.
  • Li J; Nantes Université, CHU Nantes, INSERM, Center for Research in Transplantation and Translational Immunology, UMR 1064, ITUN, 44093 Nantes, France.
  • Farrokhi V; Bamboo Therapeutics, Pfizer, Chapel Hill, NC 27514, USA.
  • Neubert H; Gene Therapy Center, Eshelman School of Pharmacy DPMP, University of North Carolina, Chapel Hill, NC 27599-7352, USA.
  • Owens J; Pfizer Inc., Andover, MA 01810, USA.
  • McIntyre M; Pfizer Inc., Andover, MA 01810, USA.
  • Moullier P; Pfizer Inc., Cambridge, MA 02139, USA.
  • Samulski RJ; Bamboo Therapeutics, Pfizer, Chapel Hill, NC 27514, USA.
Mol Ther Methods Clin Dev ; 30: 30-47, 2023 Sep 14.
Article em En | MEDLINE | ID: mdl-37746247

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: França

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: França