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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Bennett, Jean; Wellman, Jennifer; Marshall, Kathleen A; McCague, Sarah; Ashtari, Manzar; DiStefano-Pappas, Julie; Elci, Okan U; Chung, Daniel C; Sun, Junwei; Wright, J Fraser; Cross, Dominique R; Aravand, Puya; Cyckowski, Laura L; Bennicelli, Jeannette L; Mingozzi, Federico; Auricchio, Alberto; Pierce, Eric A; Ruggiero, Jason; Leroy, Bart P; Simonelli, Francesca; High, Katherine A; Maguire, Albert M.
Afiliação
  • Bennett J; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Center for Cellular and Molecular Therapeutics, The Chi
  • Wellman J; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Spark Therapeutics, Philadelphia, PA, USA.
  • Marshall KA; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • McCague S; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Ashtari M; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Department of Radiology, The Children's Hospital of Phi
  • DiStefano-Pappas J; Westat Biostatistics and Data Management Core, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Elci OU; Westat Biostatistics and Data Management Core, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Chung DC; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Spark Therapeutics, Philadelphia, PA, USA.
  • Sun J; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Wright JF; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Spark Therapeutics, Philadelphia, PA, USA.
  • Cross DR; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Aravand P; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA.
  • Cyckowski LL; Department of Radiology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Bennicelli JL; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA.
  • Mingozzi F; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Immunology and Liver Gene Therapy, Généthon, Èvry, France.
  • Auricchio A; Telethon Institute of Genetics and Medicine, Naples, Italy; Medical Genetics, Department of Pediatrics, University of Naples Federico II, Naples, Italy.
  • Pierce EA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Department of Ophthalmology, The Children's Hospital of Philadelphia, Philadelp
  • Ruggiero J; Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA.
  • Leroy BP; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Ophthalmology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Ophthalmology and Center for Medical Genetics, Ghent University Hospital and Ghen
  • Simonelli F; Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Second University of Naples, Naples, Italy.
  • High KA; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Spark Therapeutics, Philadelphia, PA, USA; Howard Hughes Medical Institute, Philadelphia, PA, USA.
  • Maguire AM; Center for Advanced Retinal and Ocular Therapeutics, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Department of Ophthalmology, Scheie Eye Institute, Univ
Lancet ; 388(10045): 661-72, 2016 Aug 13.
Article em En | MEDLINE | ID: mdl-27375040
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Visão Ocular / Terapia Genética / Cegueira / Dependovirus / Cis-trans-Isomerases / Mutação / Lobo Occipital Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male / Middle aged Idioma: En Revista: Lancet Ano de publicação: 2016 Tipo de documento: Article
Texto completo
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XML
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Visão Ocular / Terapia Genética / Cegueira / Dependovirus / Cis-trans-Isomerases / Mutação / Lobo Occipital Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Adult / Child / Female / Humans / Male / Middle aged Idioma: En Revista: Lancet Ano de publicação: 2016 Tipo de documento: Article