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

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

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

Photoreceptor function and structure in retinal degenerations caused by biallelic BEST1 mutations

由双等位基因BEST1突变引起的视网膜变性中的感光细胞功能和结构

Cideciyan, Artur V; Jacobson, Samuel G; Sumaroka, Alexander; Swider, Malgorzata; Krishnan, Arun K; Sheplock, Rebecca; Garafalo, Alexandra V; Guziewicz, Karina E; Aguirre, Gustavo D; Beltran, William A; Matsui, Yoshitsugu; Kondo, Mineo; Heon, Elise

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

Photoreceptor Function and Structure in Autosomal Dominant Vitelliform Macular Dystrophy Caused by BEST1 Mutations

由BEST1基因突变引起的常染色体显性遗传卵黄状黄斑营养不良症的光感受器功能和结构

Cideciyan, Artur V; Jacobson, Samuel G; Swider, Malgorzata; Sumaroka, Alexander; Sheplock, Rebecca; Krishnan, Arun K; Garafalo, Alexandra V; Guziewicz, Karina E; Aguirre, Gustavo D; Beltran, William A; Heon, Elise

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

Sepofarsen 1/2期试验中先天性感光细胞盲患者的视锥细胞敏感性恢复

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

Mobility test to assess functional vision in dark-adapted patients with Leber congenital amaurosis

对患有莱伯氏先天性黑蒙症的暗适应患者进行活动能力测试以评估其功能性视力。

Roman, Alejandro J; Cideciyan, Artur V; Wu, Vivian; Mascio, Abraham A; Krishnan, Arun K; Garafalo, Alexandra V; Jacobson, Samuel G

Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report

单次使用反义寡核苷酸 sepofarsen 治疗后视力得到持久改善:病例报告

Cideciyan, Artur V; Jacobson, Samuel G; Ho, Allen C; Garafalo, Alexandra V; Roman, Alejandro J; Sumaroka, Alexander; Krishnan, Arun K; Swider, Malgorzata; Schwartz, Michael R; Girach, Aniz

Childhood-onset genetic cone-rod photoreceptor diseases and underlying pathobiology

儿童期发病的遗传性视锥细胞-视杆细胞光感受器疾病及其潜在病理生物学

Garafalo, Alexandra V; Sheplock, Rebecca; Sumaroka, Alexander; Roman, Alejandro J; Cideciyan, Artur V; Jacobson, Samuel G