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A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.
Biferi, Maria Grazia; Cohen-Tannoudji, Mathilde; Cappelletto, Ambra; Giroux, Benoit; Roda, Marianne; Astord, Stéphanie; Marais, Thibaut; Bos, Corinne; Voit, Thomas; Ferry, Arnaud; Barkats, Martine.
Affiliation
  • Biferi MG; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France. Electronic address: mg.biferi@institut-myologie.org.
  • Cohen-Tannoudji M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Cappelletto A; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Giroux B; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Roda M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Astord S; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Marais T; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Bos C; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
  • Voit T; NIHR GOSH Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, London WC1N 1EH, UK.
  • Ferry A; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France; Sorbonne Paris Cité, Université Paris Descartes, Paris 75006, France.
  • Barkats M; Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.
Mol Ther ; 25(9): 2038-2052, 2017 09 06.
Article in En | MEDLINE | ID: mdl-28663100
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Dependovirus / Superoxide Dismutase-1 / Genetic Vectors / Amyotrophic Lateral Sclerosis Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Dependovirus / Superoxide Dismutase-1 / Genetic Vectors / Amyotrophic Lateral Sclerosis Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2017 Document type: Article