Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial.

Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen C; Krishnan, Arun K; Roman, Alejandro J; Garafalo, Alexandra V; Wu, Vivian; Swider, Malgorzata; Sumaroka, Alexander; Van Cauwenbergh, Caroline; Russell, Stephen R; Drack, Arlene V; Leroy, Bart P; Schwartz, Michael R; Girach, Aniz

Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen C; Krishnan, Arun K; Roman, Alejandro J; Garafalo, Alexandra V; Wu, Vivian; Swider, Malgorzata; Sumaroka, Alexander; Van Cauwenbergh, Caroline; Russell, Stephen R; Drack, Arlene V; Leroy, Bart P; Schwartz, Michael R; Girach, Aniz.

Afiliação

Cideciyan AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Jacobson SG; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Ho AC; Wills Eye Hospital, Thomas Jefferson University, Philadelphia, Pennsylvania.
Krishnan AK; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Roman AJ; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Garafalo AV; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Wu V; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Swider M; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Sumaroka A; Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Van Cauwenbergh C; Department of Ophthalmology, Ghent University and Ghent University Hospital, Ghent, Belgium.
Russell SR; The University of Iowa Institute for Vision Research, University of Iowa, Iowa City, Iowa.
Drack AV; The University of Iowa Institute for Vision Research, University of Iowa, Iowa City, Iowa.
Leroy BP; Department of Ophthalmology, Ghent University and Ghent University Hospital, Ghent, Belgium.
Schwartz MR; Center for Medical Genetics, Ghent University Hospital, Ghent, Belgium.
Girach A; Division of Ophthalmology and Center for Cellular & Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Ophthalmol Sci ; 2(2): 100133, 2022 Jun.

Article em En | MEDLINE | ID: mdl-36249682

Palavras-chave

Antisense oligonucleotides; CEP290, centrosomal protein 290; Cone photoreceptors; FST, full-field stimulus testing; Fovea; Inherited retinal degenerations; LCA, Leber congenital amaurosis; LP, light perception; Low vision; NLP, no light perception; TPLR, transient pupillary light reflex; VA, visual acuity

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Clinical_trials / Diagnostic_studies Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Clinical_trials / Diagnostic_studies Idioma: En Ano de publicação: 2022 Tipo de documento: Article