Gene therapy trial lights the way for patients with Leber congenital amaurosis 1.
作者信息
Chan Ying Kai
机构信息
Cirrus Therapeutics, Cambridge, MA, USA; Academic Unit of Ophthalmology, Translational Health Sciences, University of Bristol, BS8 1TD Bristol, UK.
出版信息
Mol Ther. 2024 Nov 6;32(11):3748-3750. doi: 10.1016/j.ymthe.2024.10.014. Epub 2024 Oct 26.
Abstract
摘要
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本文引用的文献
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Full-field Scotopic Threshold Improvement after Voretigene Neparvovec-rzyl Treatment Correlates with Chorioretinal Atrophy.经 Voretigene Neparvovec-rzyl 治疗后全视野暗点阈值改善与脉络膜视网膜萎缩相关。
Ophthalmology. 2023 Jul;130(7):764-770. doi: 10.1016/j.ophtha.2023.02.015. Epub 2023 Feb 21.
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Preclinical studies in support of phase I/II clinical trials to treat -associated Leber congenital amaurosis.支持治疗相关性莱伯先天性黑蒙的I/II期临床试验的临床前研究。
Mol Ther Methods Clin Dev. 2022 Dec 14;28:129-145. doi: 10.1016/j.omtm.2022.12.007. eCollection 2023 Mar 9.
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Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis.雷柏氏先天性黑蒙 2 型患者经脉络膜视网膜下注射 voretigene neparvovec-rzyl 后出现旁中心凹脉络膜视网膜萎缩
Ophthalmol Retina. 2022 Jan;6(1):58-64. doi: 10.1016/j.oret.2021.03.016. Epub 2021 Apr 8.
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Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses.工程化腺相关病毒载体以逃避固有免疫和炎症反应。
Sci Transl Med. 2021 Feb 10;13(580). doi: 10.1126/scitranslmed.abd3438.
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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.治疗 RPE65 介导的遗传性视网膜营养不良患者的 voretigene neparvovec（AAV2-hRPE65v2）的疗效和安全性：一项随机、对照、开放标签、3 期临床试验。
Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14.
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