Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report
Nature Medicine, vol. 27, pp. 785–789
Authors 9
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Artur V. Cideciyan corresponding
Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. artur.cideciyan@pennmedicine.upenn.edu
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Thomas Jefferson University · Wills Eye Hospital
Affiliation as printed
Wills Eye Hospital, Thomas Jefferson University, Philadelphia PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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Affiliation as printed
Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
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ProQR Therapeutics (Netherlands)
Affiliation as printed
ProQR Therapeutics, Leiden, The Netherlands
Cited by 63 stored of 64
Cited by patents worldwide 1 (Lens.org)
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Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereofUS11667913B2 2023-06-06 Active