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Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.

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Title: Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Authors: Yang P; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA. Electronic address: yangp@ohsu.edu., Pardon LP; Atsena Therapeutics, Durham, NC, USA., Ho AC; Wills Eye Hospital, Philadelphia, PA, USA., Lauer AK; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA., Yoon D; Atsena Therapeutics, Durham, NC, USA., Boye SE; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Boye SL; Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL, USA., Roman AJ; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Wu V; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Garafalo AV; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Sumaroka A; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Swider M; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Viarbitskaya I; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Aleman TS; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Pennesi ME; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA; Retina Foundation of the Southwest, Dallas, TX, USA., Kay CN; VitreoRetinal Associates, Gainesville, FL, USA., Fujita KP; Atsena Therapeutics, Durham, NC, USA., Cideciyan AV; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: cideciya@pennmedicine.upenn.edu.
Source: Lancet (London, England) [Lancet] 2024 Sep 07; Vol. 404 (10456), pp. 962-970.
Publication Type: Journal Article; Clinical Trial, Phase II; Multicenter Study; Clinical Trial, Phase I; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Country of Publication: England NLM ID: 2985213R Publication Model: Print Cited Medium: Internet ISSN: 1474-547X (Electronic) Linking ISSN: 01406736 NLM ISO Abbreviation: Lancet Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
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  Data: <searchLink fieldCode="AU" term="%22Yang+P%22">Yang P</searchLink>; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA. Electronic address: yangp@ohsu.edu.<br /><searchLink fieldCode="AU" term="%22Pardon+LP%22">Pardon LP</searchLink>; Atsena Therapeutics, Durham, NC, USA.<br /><searchLink fieldCode="AU" term="%22Ho+AC%22">Ho AC</searchLink>; Wills Eye Hospital, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Lauer+AK%22">Lauer AK</searchLink>; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA.<br /><searchLink fieldCode="AU" term="%22Yoon+D%22">Yoon D</searchLink>; Atsena Therapeutics, Durham, NC, USA.<br /><searchLink fieldCode="AU" term="%22Boye+SE%22">Boye SE</searchLink>; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Boye+SL%22">Boye SL</searchLink>; Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Roman+AJ%22">Roman AJ</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Wu+V%22">Wu V</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Garafalo+AV%22">Garafalo AV</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Sumaroka+A%22">Sumaroka A</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Swider+M%22">Swider M</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Viarbitskaya+I%22">Viarbitskaya I</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Aleman+TS%22">Aleman TS</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Pennesi+ME%22">Pennesi ME</searchLink>; Casey Eye Institute, Oregon Health & Science University, Portland, OR, USA; Retina Foundation of the Southwest, Dallas, TX, USA.<br /><searchLink fieldCode="AU" term="%22Kay+CN%22">Kay CN</searchLink>; VitreoRetinal Associates, Gainesville, FL, USA.<br /><searchLink fieldCode="AU" term="%22Fujita+KP%22">Fujita KP</searchLink>; Atsena Therapeutics, Durham, NC, USA.<br /><searchLink fieldCode="AU" term="%22Cideciyan+AV%22">Cideciyan AV</searchLink>; Center for Hereditary Retinal Degenerations, Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: cideciya@pennmedicine.upenn.edu.
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  Data: <searchLink fieldCode="JN" term="%222985213R%22">Lancet (London, England)</searchLink> [Lancet] 2024 Sep 07; Vol. 404 (10456), pp. 962-970.
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