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Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I.
Cataldi, Marcela P; Vannoy, Charles H; Blaeser, Anthony; Tucker, Jason D; Leroy, Victoria; Rawls, Raegan; Killilee, Jessalyn; Holbrook, Molly C; Lu, Qi Long.
Affiliation
  • Cataldi MP; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA. Electronic address: marcela.cataldi@atriumhealth.org.
  • Vannoy CH; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Blaeser A; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Tucker JD; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Leroy V; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Rawls R; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Killilee J; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Holbrook MC; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA.
  • Lu QL; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC 28203, USA. Electronic address: qi.lu@atriumhealth.org.
Mol Ther ; 31(12): 3478-3489, 2023 Dec 06.
Article de En | MEDLINE | ID: mdl-37919902

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Pentosyltransferases / Dystrophies musculaires Limites: Animals / Humans Langue: En Journal: Mol Ther Sujet du journal: BIOLOGIA MOLECULAR / TERAPEUTICA Année: 2023 Type de document: Article Pays de publication: États-Unis d'Amérique

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Pentosyltransferases / Dystrophies musculaires Limites: Animals / Humans Langue: En Journal: Mol Ther Sujet du journal: BIOLOGIA MOLECULAR / TERAPEUTICA Année: 2023 Type de document: Article Pays de publication: États-Unis d'Amérique