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

Exploring extracellular vesicle MicroRNAs in Usher syndrome type 1B: Tear-Derived EVs as potential indicators of retinal health

探索 Usher 综合征 1B 型中的细胞外囊泡微 RNA:泪液来源的细胞外囊泡作为视网膜健康的潜在指标

Bervoets, Sander; Duijkers, Lonneke; Velde, Hedwig M; Corradi, Zelia; van Oosten, Edwin M; Suárez-Herrera, Nuria; Garanto, Alejandro; Moreno-Pelayo, Miguel A; Pennings, Ronald J E; Collin, Rob W J; Vázquez-Domínguez, Irene

A Comparison of Randomizing Either One Eye or Both Eyes in Clinical Trials for Stargardt Disease Type 1

Stargardt病1型临床试验中单眼或双眼随机分组的比较

Pas, Jeroen A A H; Dhooge, Patty P A; Li, Catherina H Z; Collin, Rob W J; Hoyng, Carel B; IntHout, Joanna

Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoids

小分子治疗可缓解LCA5缺陷型人类视网膜类器官的光感受器纤毛缺陷

Athanasiou, Dimitra; Afanasyeva, Tess A V; Chai, Niuzheng; Ziaka, Kalliopi; Jovanovic, Katarina; Guarascio, Rosellina; Boldt, Karsten; Corral-Serrano, Julio C; Kanuga, Naheed; Roepman, Ronald; Collin, Rob W J; Cheetham, Michael E

Correction: Small molecule treatment alleviates photoreceptor cilia defects in LCA5-deficient human retinal organoids

更正:小分子治疗可缓解LCA5缺陷型人类视网膜类器官的光感受器纤毛缺陷

Athanasiou, Dimitra; Afanasyeva, Tess A V; Chai, Niuzheng; Ziaka, Kalliopi; Jovanovic, Katarina; Guarascio, Rosellina; Boldt, Karsten; Corral-Serrano, Julio C; Kanuga, Naheed; Roepman, Ronald; Collin, Rob W J; Cheetham, Michael E

Progression of Atrophy as a Function of ABCA4 Variants and Age of Onset in Stargardt Disease

Stargardt病中萎缩进展与ABCA4变异和发病年龄的关系

Pas, Jeroen A A H; Li, Catherina H Z; Van den Broeck, Filip; Dhooge, Patty P A; De Zaeytijd, Julie; Collin, Rob W J; Leroy, Bart P; Hoyng, Carel B

Actigraphy-based assessment of circadian rhythmicity and sleep in patients with Usher syndrome type 2a: A case-control study

基于活动记录仪的 Usher 综合征 2a 型患者昼夜节律和睡眠评估:一项病例对照研究

Hendricks, Jessie M; Metz, Juriaan R; Boss, H Myrthe; Collin, Rob W J; de Vrieze, Erik; van Wijk, Erwin

Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease

针对 Stargardt 病中 ABCA4 变异 c.768G>T 的剪接调节疗法的临床前评估

Dyah W Karjosukarso, Femke Bukkems, Lonneke Duijkers, Tomasz Z Tomkiewicz, Julia Kiefmann, Andrei Sarlea, Sander Bervoets, Irene Vázquez-Domínguez, Laurie L Molday, Robert S Molday, Mihai G Netea, Carel B Hoyng, Alejandro Garanto, Rob W J Collin

Efficacy, biodistribution and safety comparison of chemically modified antisense oligonucleotides in the retina

化学修饰反义寡核苷酸在视网膜中的疗效、生物分布和安全性比较

Vázquez-Domínguez, Irene; Anido, Alejandro Allo; Duijkers, Lonneke; Hoppenbrouwers, Tamara; Hoogendoorn, Anita D M; Koster, Céline; Collin, Rob W J; Garanto, Alejandro

Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4

利用单个 U7snRNA 载体递送多个 AON 以恢复 ABCA4 剪接缺陷的概念验证

Suárez-Herrera, Nuria; Riswick, Iris B; Vázquez-Domínguez, Irene; Duijkers, Lonneke; Karjosukarso, Dyah W; Piccolo, Davide; Bauwens, Miriam; De Baere, Elfride; Cheetham, Michael E; Garanto, Alejandro; Collin, Rob W J

In vivo genome editing for inherited retinal disease: Opportunities and challenges

利用体内基因组编辑技术治疗遗传性视网膜疾病:机遇与挑战

Collin, Rob W J; Leroy, Bart P