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

Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness

通过 AAV 基因治疗靶向 ON-双极细胞可稳定逆转 LRIT3-先天性静止性夜盲症

Keiko Miyadera, Evelyn Santana, Karolina Roszak, Sommer Iffrig, Meike Visel, Simone Iwabe, Ryan F Boyd, Joshua T Bartoe, Yu Sato, Alexa Gray, Ana Ripolles-Garcia, Valérie L Dufour, Leah C Byrne, John G Flannery, William A Beltran, Gustavo D Aguirre

scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution

scAAVengr,一种基于转录组的流程,用于以单细胞分辨率对工程化 AAV 进行定量排序

Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Luis Felipe L Pompeo Marinho, Gustavo D Aguirre, José-Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R

Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy

后期 RPE65 基因治疗的长期结构性结果

Kristin L Gardiner, Artur V Cideciyan, Malgorzata Swider, Valérie L Dufour, Alexander Sumaroka, András M Komáromy, William W Hauswirth, Simone Iwabe, Samuel G Jacobson, William A Beltran, Gustavo D Aguirre

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

Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology

与 RPGRIP1 相关的视锥-视杆营养不良症犬模型中视网膜功能和结构的变化支持多基因病因

Rueben G Das, Felipe Pompeo Marinho, Simone Iwabe, Evelyn Santana, Kendra Sierra McDaid, Gustavo D Aguirre, Keiko Miyadera

Assessment of visual function and retinal structure following acute light exposure in the light sensitive T4R rhodopsin mutant dog

光敏感 T4R 视紫红质突变犬急性光照后的视觉功能和视网膜结构评估

Simone Iwabe, Gui-Shuang Ying, Gustavo D Aguirre, William A Beltran

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease

成功抑制光感受器和视力丧失可将视网膜基因治疗的治疗窗口扩展到疾病的后期

William A Beltran, Artur V Cideciyan, Simone Iwabe, Malgorzata Swider, Mychajlo S Kosyk, Kendra McDaid, Inna Martynyuk, Gui-Shuang Ying, James Shaffer, Wen-Tao Deng, Sanford L Boye, Alfred S Lewin, William W Hauswirth, Samuel G Jacobson, Gustavo D Aguirre

Exclusion of RPGRIP1 ins44 from primary causal association with early-onset cone-rod dystrophy in dogs

将 RPGRIP1 ins44 排除在与犬早发性视锥-视杆营养不良症的主要因果关系之外

Tatyana Kuznetsova, Simone Iwabe, Kathleen Boesze-Battaglia, Sue Pearce-Kelling, Yim Chang-Min, Kendra McDaid, Keiko Miyadera, Andras Komaromy, Gustavo D Aguirre