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Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia.
Lee, Young Mok; Conlon, Thomas J; Specht, Andrew; Coleman, Kirsten E; Brown, Laurie M; Estrella, Ana M; Dambska, Monika; Dahlberg, Kathryn R; Weinstein, David A.
Afiliação
  • Lee YM; Glycogen Storage Disease Program, Department of Pediatrics, University of Connecticut School of Medicine, Farmington, CT, 06030, USA.
  • Conlon TJ; Glycogen Storage Disease Program, University of Florida College of Medicine, Gainesville, FL, USA.
  • Specht A; Powell Gene Therapy Center, University of Florida, Gainesville, FL, 32610, USA.
  • Coleman KE; CR Scientific and Compliance Consulting, LLC, Gainesville, FL, 32608, USA.
  • Brown LM; Department of Small Animal Clinical Sciences, University of Florida College of Veterinary Medicine, Gainesville, FL, USA.
  • Estrella AM; Powell Gene Therapy Center, University of Florida, Gainesville, FL, 32610, USA.
  • Dambska M; Glycogen Storage Disease Program, University of Florida College of Medicine, Gainesville, FL, USA.
  • Dahlberg KR; Glycogen Storage Disease Program, Department of Pediatrics, University of Connecticut School of Medicine, Farmington, CT, 06030, USA.
  • Weinstein DA; Glycogen Storage Disease Program, University of Florida College of Medicine, Gainesville, FL, USA.
J Inherit Metab Dis ; 41(6): 977-984, 2018 11.
Article em En | MEDLINE | ID: mdl-29802554
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Doença de Depósito de Glicogênio Tipo I Limite: Animals País/Região como assunto: Europa Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2018 Tipo de documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Doença de Depósito de Glicogênio Tipo I Limite: Animals País/Região como assunto: Europa Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2018 Tipo de documento: Article