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

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

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

A Novel ARL3 Gene Mutation Associated With Autosomal Dominant Retinal Degeneration.

一种与常染色体显性视网膜变性相关的新型 ARL3 基因突变

Ratnapriya Rinki, Jacobson Samuel G, Cideciyan Artur V, English Milton A, Roman Alejandro J, Sumaroka Alexander, Sheplock Rebecca, Swaroop Anand

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

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; Cheang, Bee-Lin; Davidson, Vanessa; O'Riordan, Catherine R; Dizhoor, Alexander M; Boye, Shannon E

Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives

遗传性视网膜疾病治疗进展:早期视网膜下基因治疗临床试验及未来计划候选方案

Garafalo, Alexandra V; Cideciyan, Artur V; Héon, Elise; Sheplock, Rebecca; Pearson, Alexander; WeiYang Yu, Caberry; Sumaroka, Alexander; Aguirre, Gustavo D; Jacobson, Samuel G

Foveal Therapy in Blue Cone Monochromacy: Predictions of Visual Potential From Artificial Intelligence

蓝锥单色视觉中的中心凹治疗:基于人工智能的视觉潜能预测

Sumaroka, Alexander; Cideciyan, Artur V; Sheplock, Rebecca; Wu, Vivian; Kohl, Susanne; Wissinger, Bernd; Jacobson, Samuel G

Treatment Potential for Macular Cone Vision in Leber Congenital Amaurosis Due to CEP290 or NPHP5 Mutations: Predictions From Artificial Intelligence

人工智能预测由 CEP290 或 NPHP5 基因突变引起的 Leber 先天性黑蒙患者的黄斑锥体视力障碍的治疗潜力

Sumaroka, Alexander; Garafalo, Alexandra V; Semenov, Evelyn P; Sheplock, Rebecca; Krishnan, Arun K; Roman, Alejandro J; Jacobson, Samuel G; Cideciyan, Artur V