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Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report.
Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen C; Garafalo, Alexandra V; Roman, Alejandro J; Sumaroka, Alexander; Krishnan, Arun K; Swider, Malgorzata; Schwartz, Michael R; Girach, Aniz.
Afiliação
  • Cideciyan AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. artur.cideciyan@pennmedicine.upenn.edu.
  • Jacobson SG; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Ho AC; Wills Eye Hospital, Thomas Jefferson University, Philadelphia PA, USA.
  • Garafalo AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Roman AJ; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Sumaroka A; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Krishnan AK; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Swider M; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Schwartz MR; ProQR Therapeutics, Leiden, The Netherlands.
  • Girach A; ProQR Therapeutics, Leiden, The Netherlands.
Nat Med ; 27(5): 785-789, 2021 05.
Article em En | MEDLINE | ID: mdl-33795869
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Oligonucleotídeos Antissenso / Proteínas de Ciclo Celular / Proteínas do Citoesqueleto / Amaurose Congênita de Leber / Ciliopatias / Antígenos de Neoplasias Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Oligonucleotídeos Antissenso / Proteínas de Ciclo Celular / Proteínas do Citoesqueleto / Amaurose Congênita de Leber / Ciliopatias / Antígenos de Neoplasias Limite: Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article