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

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

在犬X连锁视网膜色素变性模型中,通过视网膜下注射递送表达密码子优化RPGR的腺相关病毒载体的毒性和疗效评价

Dufour, Valérie L; Cideciyan, Artur V; Ye, Guo-Jie; Song, Chunjuan; Timmers, Adrian; Habecker, Perry L; Pan, Wei; Weinstein, Nicole M; Swider, Malgorzata; Durham, Amy C; Ying, Gui-Shuang; Robinson, Paulette M; Jacobson, Samuel G; Knop, David R; Chulay, Jeffrey D; Shearman, Mark S; Aguirre, Gustavo D; Beltran, William A

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

在接受视网膜下基因治疗的X连锁视网膜色素变性犬模型中,使用两种RPGR转基因进行剂量范围探索研究

Song, Chunjuan; Dufour, Valérie L; Cideciyan, Artur V; Ye, Guo-Jie; Swider, Malgorzata; Newmark, Judith A; Timmers, Adrian M; Robinson, Paulette M; Knop, David R; Chulay, Jeffrey D; Jacobson, Samuel G; Aguirre, Gustavo D; Beltran, William A; Shearman, Mark S

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

rAAV 从肌肉注射到肢体灌注输送的衔接:非人类灵长类动物研究中的优化

Alisha M Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R Knop, Gensheng Wang, Janet Benson, Kristen E Coleman, Allison M Keeler, Christian Mueller, Louis G Chicoine, Jeffrey D Chulay, Terence R Flotte

Prospective Evaluation of Patients With X-Linked Retinoschisis During 18 Months

对X连锁视网膜劈裂症患者进行为期18个月的前瞻性评估

Pennesi, Mark E; Birch, David G; Jayasundera, K Thiran; Parker, Maria; Tan, Ou; Gurses-Ozden, Rabia; Reichley, Carrie; Beasley, Kathleen N; Yang, Paul; Weleber, Richard G; Bennett, Lea D; Heckenlively, John R; Kothapalli, Kalyani; Chulay, Jeffrey D; For The Xlrs-Study Group

Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy

RPE65缺陷型视网膜营养不良基因治疗5年后的结果

Pennesi, Mark E; Weleber, Richard G; Yang, Paul; Whitebirch, Chris; Thean, Beverly; Flotte, Terence R; Humphries, Margaret; Chegarnov, Elvira; Beasley, Kathleen N; Stout, J Timothy; Chulay, Jeffrey D

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

在RPGR缺陷型Rd9小鼠中,表达密码子优化RPGR的AAV载体的毒理学和药理学研究

Song, Chunjuan; Conlon, Thomas J; Deng, Wen-Tao; Coleman, Kirsten E; Zhu, Ping; Plummer, Cayrn; Mandapati, Savitri; Van Hoosear, Mailin; Green, Kari B; Sonnentag, Peter; Sharma, Alok K; Timmers, Adrian; Robinson, Paulette M; Knop, David R; Hauswirth, William W; Chulay, Jeffrey D; Shearman, Mark S; Ye, Guo-Jie

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

优化RPGR基因突变引起的X连锁视网膜色素变性的视网膜基因治疗

William A Beltran,Artur V Cideciyan,Shannon E Boye,Guo-Jie Ye,Simone Iwabe,Valerie L Dufour,Luis Felipe Marinho,Malgorzata Swider,Mychajlo S Kosyk,Jin Sha,Sanford L Boye,James J Peterson,C Douglas Witherspoon,John J Alexander,Gui-Shuang Ying,Mark S Shearman,Jeffrey D Chulay,William W Hauswirth,Paul D Gamlin,Samuel G Jacobson,Gustavo D Aguirre

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

α1-抗胰蛋白酶缺乏症基因治疗后5年表达和中性粒细胞缺陷修复

Mueller, Christian; Gernoux, Gwladys; Gruntman, Alisha M; Borel, Florie; Reeves, Emer P; Calcedo, Roberto; Rouhani, Farshid N; Yachnis, Anthony; Humphries, Margaret; Campbell-Thompson, Martha; Messina, Louis; Chulay, Jeffrey D; Trapnell, Bruce; Wilson, James M; McElvaney, Noel G; Flotte, Terence R

Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs

表达人或犬 CNGB3 的 AAV5 载体在 CNGB3 突变犬中的安全性和有效性

Ye, Guo-Jie; Komáromy, András M; Zeiss, Caroline; Calcedo, Roberto; Harman, Christine D; Koehl, Kristin L; Stewart, Gabriel A; Iwabe, Simone; Chiodo, Vince A; Hauswirth, William W; Aguirre, Gustavo D; Chulay, Jeffrey D

Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia

CNGB3相关性全色盲中残余中央凹锥体结构

Langlo, Christopher S; Patterson, Emily J; Higgins, Brian P; Summerfelt, Phyllis; Razeen, Moataz M; Erker, Laura R; Parker, Maria; Collison, Frederick T; Fishman, Gerald A; Kay, Christine N; Zhang, Jing; Weleber, Richard G; Yang, Paul; Wilson, David J; Pennesi, Mark E; Lam, Byron L; Chiang, John; Chulay, Jeffrey D; Dubra, Alfredo; Hauswirth, William W; Carroll, Joseph