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CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy.
Geoffroy, Marine; Pili, Louna; Buffa, Valentina; Caroff, Maëlle; Bigot, Anne; Gicquel, Evelyne; Rouby, Grégory; Richard, Isabelle; Fragnoud, Romain.
Affiliation
  • Geoffroy M; Généthon, 91000 Evry-Courcouronnes, France.
  • Pili L; Université Paris-Saclay/Université Evry, INSERM, Généthon, Integrare Research Unit, UMR_S951, 91000 Evry, France.
  • Buffa V; Généthon, 91000 Evry-Courcouronnes, France.
  • Caroff M; Université Paris-Saclay/Université Evry, INSERM, Généthon, Integrare Research Unit, UMR_S951, 91000 Evry, France.
  • Bigot A; Généthon, 91000 Evry-Courcouronnes, France.
  • Gicquel E; Université Paris-Saclay/Université Evry, INSERM, Généthon, Integrare Research Unit, UMR_S951, 91000 Evry, France.
  • Rouby G; Généthon, 91000 Evry-Courcouronnes, France.
  • Richard I; Université Paris-Saclay/Université Evry, INSERM, Généthon, Integrare Research Unit, UMR_S951, 91000 Evry, France.
  • Fragnoud R; Institut de Myologie, Université Pierre et Marie Curie Paris 6, UM76 Univ. Paris 6/U974 UMR7215, CNRS Pitié-Salpétrière-INSERM, UMRS 974, 75000 Paris, France.
Cells ; 12(20)2023 10 12.
Article in En | MEDLINE | ID: mdl-37887288

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pentosyltransferases / Muscular Dystrophies, Limb-Girdle Limits: Humans Language: En Journal: Cells Year: 2023 Document type: Article Affiliation country:

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pentosyltransferases / Muscular Dystrophies, Limb-Girdle Limits: Humans Language: En Journal: Cells Year: 2023 Document type: Article Affiliation country: