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Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
Aleman, Tomas S; Huckfeldt, Rachel M; Serrano, Leona W; Pearson, Denise J; Vergilio, Grace K; McCague, Sarah; Marshall, Kathleen A; Ashtari, Manzar; Doan, Tu M; Weigel-DiFranco, Carol A; Biron, Bethany S; Wen, Xiao-Hong; Chung, Daniel C; Liu, Emily; Ferenchak, Kevin; Morgan, Jessica I W; Pierce, Eric A; Eliott, Dean; Bennett, Jean; Comander, Jason; Maguire, Albert M.
Afiliación
  • Aleman TS; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania; The Children's Hospital of Philadelphia, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsy
  • Huckfeldt RM; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Serrano LW; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania.
  • Pearson DJ; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania.
  • Vergilio GK; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania.
  • McCague S; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania; The Children's Hospital of Philadelphia, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Marshall KA; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania; The Children's Hospital of Philadelphia, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Ashtari M; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania; The Children's Hospital of Philadelphia, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania.
  • Doan TM; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Weigel-DiFranco CA; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Biron BS; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Wen XH; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Chung DC; Spark Therapeutics Inc., Philadelphia, Pennsylvania.
  • Liu E; Spark Therapeutics Inc., Philadelphia, Pennsylvania.
  • Ferenchak K; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Morgan JIW; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania.
  • Pierce EA; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Eliott D; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Bennett J; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania.
  • Comander J; Ocular Genomics Institute, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts.
  • Maguire AM; Scheie Eye Institute at the Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania; The Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania; The Children's Hospital of Philadelphia, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsy
Ophthalmology ; 129(10): 1177-1191, 2022 10.
Article en En | MEDLINE | ID: mdl-35714735
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Perforaciones de la Retina / Coroideremia Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline / Observational_studies / Risk_factors_studies Límite: Adult / Humans / Middle aged Idioma: En Revista: Ophthalmology Año: 2022 Tipo del documento: Article
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Perforaciones de la Retina / Coroideremia Tipo de estudio: Clinical_trials / Diagnostic_studies / Guideline / Observational_studies / Risk_factors_studies Límite: Adult / Humans / Middle aged Idioma: En Revista: Ophthalmology Año: 2022 Tipo del documento: Article