日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial

基因治疗后,莱伯西林相关性视网膜纤毛病患者视锥细胞介导的视觉功能恢复:一项 I/II 期试验的一年结果

Aleman, Tomas S; Uyhazi, Katherine E; Roman, Alejandro J; Weber, Mariejel L; O'Neil, Erin C; Swider, Malgorzata; Sumaroka, Alexander; Maguire, Katherine H; Aleman, Elena M; Santos, Arlene J; Kim, Rebecca J; Parchinski, Kelsey M; Billek, Andrew; Fradin, Makayla; Chung, William; Margaritis, Paris; Sun, Junwei; Scoles, Drew H; Wu, Vivian; Garafalo, Alexandra V; Jayagopal, Ashwath; Yerxa, Ben; Tuller, Sarah; Maguire, Albert M; Bennett, Jean; Cideciyan, Artur V

Measuring Rod- and Cone-Photoreceptor-Specific Vision in Inherited Retinal Diseases Using a Commercial Perimeter

使用商用视野计测量遗传性视网膜疾病患者的视杆细胞和视锥细胞特异性视力

Wu, Vivian; Roman, Alejandro J; Galsterer, Emma L; Ansari, Georg; Erdinest, Inbar; Righetti, Giulia; Viarbitskaya, Iryna; Russell, Robert C; Kim, Rebecca J; Charlier, Jacques; Pfau, Kristina; Stingl, Krunoslav; Banin, Eyal; Pfau, Maximilian; Stingl, Katarina; Aleman, Tomas S; Cideciyan, Artur V

Location and Identity of Photoreceptors Contributing to the Full-Field Stimulus Test (FST)

参与全视野刺激测试 (FST) 的光感受器的位置和类型

Cideciyan, Artur V; Neuhauser, Hongsong; Roman, Alejandro J; Aleman, Tomas S

Evaluation of Retinal Structure and Visual Function in Blue Cone Monochromacy to Develop Clinical Endpoints for L-opsin Gene Therapy

评估蓝锥单色视患者的视网膜结构和视觉功能,以建立L-视蛋白基因治疗的临床终点

Cideciyan, Artur V; Roman, Alejandro J; Warner, Raymond L; Sumaroka, Alexander; Wu, Vivian; Jiang, Yu Y; Swider, Malgorzata; Garafalo, Alexandra V; Viarbitskaya, Iryna; Russell, Robert C; Kohl, Susanne; Wissinger, Bernd; Ripamonti, Caterina; Barbur, John L; Bach, Michael; Carroll, Joseph; Morgan, Jessica I W; Aleman, Tomas S

Retinal Degeneration Associated With Biallelic RDH12 Variants: Longitudinal Evaluation of Retinal Structure and Visual Function in Pediatric Patients

与RDH12双等位基因变异相关的视网膜变性:儿科患者视网膜结构和视觉功能的纵向评估

Aleman, Tomas S; Roman, Alejandro J; Uyhazi, Katherine E; Jiang, Yu You; Bedoukian, Emma C; Sumaroka, Alexander; Wu, Vivian; Swider, Malgorzata; Viarbitskaya, Iryna; Russell, Robert C; Shagena, Elizabeth O; Santos, Arlene J; Serrano, Leona W; Parchinski, Kelsey M; Kim, Rebecca J; Weber, Mariejel L; Garafalo, Alexandra V; Thompson, Dorothy A; Maguire, Albert M; Bennett, Jean; Scoles, Drew H; O'Neil, Erin C; Morgan, Jessica I W; Cideciyan, Artur V

Erratum: Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

更正:基因疗法治疗由GUCY2D突变引起的儿童失明后,安全性和疗效均有所提高

Jacobson, Samuel G; Cideciyan, Artur V; Ho, Allen C; Peshenko, Igor V; Garafalo, Alexandra V; Roman, Alejandro J; Sumaroka, Alexander; Wu, Vivian; Krishnan, Arun K; Sheplock, Rebecca; Boye, Sanford L; Dizhoor, Alexander M; Boye, Shannon E

Treatment Strategy With Gene Editing for Late-Onset Retinal Degeneration Caused by a Founder Variant in C1QTNF5

利用基因编辑治疗由C1QTNF5基因创始变异引起的迟发性视网膜变性

Li, Randa T H; Roman, Alejandro J; Sumaroka, Alexander; Stanton, Chloe M; Swider, Malgorzata; Garafalo, Alexandra V; Heon, Elise; Vincent, Ajoy; Wright, Alan F; Megaw, Roly; Aleman, Tomas S; Browning, Andrew C; Dhillon, Baljean; Cideciyan, Artur V

Color Vision in Blue Cone Monochromacy: Outcome Measures for a Clinical Trial

蓝锥单色视觉的色觉:临床试验的结果指标

Mascio, Abraham A; Roman, Alejandro J; Cideciyan, Artur V; Sheplock, Rebecca; Wu, Vivian; Garafalo, Alexandra V; Sumaroka, Alexander; Pirkle, Sydney; Kohl, Susanne; Wissinger, Bernd; Jacobson, Samuel G; Barbur, John L

Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes

CEP290-LCA 患者单次玻璃体内注射反义寡核苷酸后视力得到持久改善:双眼验证

Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen C; Swider, Malgorzata; Sumaroka, Alexander; Roman, Alejandro J; Wu, Vivian; Russell, Robert C; Viarbitskaya, Iryna; Garafalo, Alexandra V; Schwartz, Michael R; Girach, Aniz

Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

玻璃体内注射反义寡核苷酸 sepofarsen 治疗 Leber 先天性黑蒙 10 型:一项 1b/2 期试验

Russell, Stephen R; Drack, Arlene V; Cideciyan, Artur V; Jacobson, Samuel G; Leroy, Bart P; Van Cauwenbergh, Caroline; Ho, Allen C; Dumitrescu, Alina V; Han, Ian C; Martin, Mitchell; Pfeifer, Wanda L; Sohn, Elliott H; Walshire, Jean; Garafalo, Alexandra V; Krishnan, Arun K; Powers, Christian A; Sumaroka, Alexander; Roman, Alejandro J; Vanhonsebrouck, Eva; Jones, Eltanara; Nerinckx, Fanny; De Zaeytijd, Julie; Collin, Rob W J; Hoyng, Carel; Adamson, Peter; Cheetham, Michael E; Schwartz, Michael R; den Hollander, Wilhelmina; Asmus, Friedrich; Platenburg, Gerard; Rodman, David; Girach, Aniz