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

Safety and vision outcomes of subretinal gene supplementation therapy in PDE6A-associated retinitis pigmentosa: a non-randomised controlled trial

视网膜下基因补充疗法治疗PDE6A相关性视网膜色素变性的安全性和视力结果:一项非随机对照试验

Reichel, Felix F; Fischer, M Dominik; Stingl, Katarina; Kuehlewein, Laura; Seitz, Immanuel; Peters, Tobias; Ziegler, Melanie; Wilhelm, Barbara; Kohl, Susanne; Weisschuh, Nicole; Martus, Peter; Seeliger, Mathias; Muehlfriedel, Regine; Paquet-Durand, Francois; Tsang, Stephen H; Bartz-Schmidt, Karl Ulrich; Ueffing, Marius; Zrenner, Eberhart; Biel, Martin; Wissinger, Bernd; Michalakis, Stylianos

Ocular Safety and Toxicology of Subretinal Gene Therapy With rAAV.hPDE6A in Nonhuman Primates

非人灵长类动物视网膜下rAAV.hPDE6A基因治疗的眼部安全性和毒理学研究

Seitz, Immanuel P; Wozar, Fabian; Ochakovski, Guy Alex; Reichel, Felix F; Korte, Sven; Korbmacher, Birgit; Wilhelm, Barbara; Süsskind, Daniela; Bartz-Schmidt, Karl-Ulrich; Fischer, M Dominik; Peters, Tobias

dCasMINI-mediated therapy rescues photoreceptors degeneration in a mouse model of retinitis pigmentosa

dCasMINI 介导的疗法挽救了视网膜色素变性小鼠模型中的光感受器退化

Qing Wang, Xiaoshu Xu, Siyu Chen, Rui Lu, Liang Li, Chien-Hui Lo, Zhiquan Liu, Ke Ning, Tingting Li, Tia J Kowal, Biao Wang, Mary E Hartnett, Sui Wang, Lei S Qi, Yang Sun

Roles of transcriptional factor PsrA in the regulation of quorum sensing in Pseudomonas aeruginosa PAO1

转录因子PsrA在铜绿假单胞菌PAO1中群体感应的调控作用

Li-Ching Kok, Chia-Chun Tsai, Yu-Hsuan Liao, Yi-Ling Lo, Nai-Wei Cheng, Ching-Ting Lin, Hwan-You Chang

Efficient Rescue of Retinal Degeneration in Pde6a Mice by Engineered Base Editing and Prime Editing

通过工程化碱基编辑和 Prime 编辑有效挽救 Pde6a 小鼠的视网膜变性

Zhiquan Liu, Siyu Chen, Alexander E Davis, Chien-Hui Lo, Qing Wang, Tingting Li, Ke Ning, Qi Zhang, Jingyu Zhao, Sui Wang, Yang Sun

Central Visual Function and Genotype-Phenotype Correlations in PDE6A-Associated Retinitis Pigmentosa

PDE6A相关性视网膜色素变性患者的中心视觉功能与基因型-表型相关性

Kuehlewein, Laura; Straßer, Torsten; Blumenstock, Gunnar; Stingl, Katarina; Fischer, M Dominik; Wilhelm, Barbara; Zrenner, Eberhart; Wissinger, Bernd; Kohl, Susanne; Weisschuh, Nicole; Zobor, Ditta

Novel variants in PDE6A and PDE6B genes and its phenotypes in patients with retinitis pigmentosa in Chinese families

中国家族性视网膜色素变性患者中PDE6A和PDE6B基因的新变异及其表型

Li, Yuyu; Li, Ruyi; Dai, Hehua; Li, Genlin

Novel mutations in PDE6A and CDHR1 cause retinitis pigmentosa in Pakistani families

PDE6A 和 CDHR1 基因的新突变导致巴基斯坦家族出现视网膜色素变性

Dawood, Muhammad; Lin, Siying; Din, Taj Ud; Shah, Irfan Ullah; Khan, Niamat; Jan, Abid; Marwan, Muhammad; Sultan, Komal; Nowshid, Maha; Tahir, Raheel; Ahmed, Asif Naveed; Yasin, Muhammad; Baple, Emma L; Crosby, Andrew H; Saleha, Shamim

Clinical Phenotype and Course of PDE6A-Associated Retinitis Pigmentosa Disease, Characterized in Preparation for a Gene Supplementation Trial

PDE6A相关性视网膜色素变性疾病的临床表型和病程特征分析,为基因补充试验做准备

Kuehlewein, Laura; Zobor, Ditta; Andreasson, Sten Olof; Ayuso, Carmen; Banfi, Sandro; Bocquet, Beatrice; Bernd, Antje S; Biskup, Saskia; Boon, Camiel J F; Downes, Susan M; Fischer, M Dominik; Holz, Frank G; Kellner, Ulrich; Leroy, Bart P; Meunier, Isabelle; Nasser, Fadi; Rosenberg, Thomas; Rudolph, Günther; Stingl, Katarina; Thiadens, Alberta A H J; Wilhelm, Barbara; Wissinger, Bernd; Zrenner, Eberhart; Kohl, Susanne; Weisschuh, Nicole

Moving Towards PDE6A Gene Supplementation Therapy

迈向PDE6A基因补充疗法

Bujakowska, Kinga M; Comander, Jason