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

Laterally spreading AAV.SPR enables safe and efficient RS1 delivery to the macula after peripheral subretinal injection

横向扩散的AAV.SPR可在周边视网膜下注射后安全有效地将RS1递送至黄斑。

Andres-Mateos, Eva; Kozlowski, Christopher; Zhang, Hangning; Fajardo, Diego; Xu, Emily; Enamorado, Ignacio Navas; Calabro, Kaitlyn R; Rayaprolu, Sruti; Havlik, L Patrick; Handy, Tess E; Plummer, Caryn E; Stevenson, Victoria; Rind, Howard; Leahy, Matthew; Boyd, Ryan F; Coleman, Kirsten E; Boye, Sanford L; Boye, Shannon E

A novel multiplex RNAi therapy simultaneously targets Hif1a and Hif2a to defy retinal degeneration in two models of AMD

一种新型多重RNAi疗法可同时靶向Hif1a和Hif2a,从而在两种AMD模型中阻止视网膜退化。

Ebner, Lynn J A; Imsand, Cornelia; Karademir, Duygu; Peters, Florian; Kiessling, Eva; Fottner, Antonia; Matter, Claudia; Fajardo, Diego S; Merolla, Luca; Wögenstein, Gabriele M; Tsioti, Ioanna; Govers, Larissa P; Blaser, Frank; Meneau, Isabelle; Boye, Sanford L; Boye, Shannon E; Grimm, Christian; Samardzija, Marijana

Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.

开发基于 AAV 的基因疗法治疗弗里德赖希共济失调的眼部表型。

Tang Heyu, Gupte Siddhant, Xu Emily, Calabro Kaitlyn R, Friend Hannah, Crosson Sean M, Fajardo Diego, Kostamo Zachary, Zhang Hangning, Peterson James J, Lin Fangyu, Kozmik Zbynek, Lutz Cathleen M, Boye Sanford L, Boye Shannon E

Current clinical applications of AAV-mediated gene therapy

目前AAV介导的基因治疗的临床应用

Byrne, Barry J; Flanigan, Kevin M; Matesanz, Susan E; Finkel, Richard S; Waldrop, Megan A; D'Ambrosio, Eleonora S; Johnson, Nicholas E; Smith, Barbara K; Bönnemann, Carsten; Carrig, Sean; Rossano, Joseph W; Greenberg, Barry; Lalaguna, Laura; Lara-Pezzi, Enrique; Subramony, Sub; Corti, Manuela; Mercado-Rodriguez, Claudia; Leon-Astudillo, Carmen; Ahrens-Nicklas, Rebecca; Bharucha-Goebel, Diana; Gao, Guangping; Gessler, Dominic J; Hwu, Wuh-Liang; Chien, Yin-Hsiu; Lee, Ni-Chung; Boye, Sanford L; Boye, Shannon E; George, Lindsey A

Assessment of the Orbit® subretinal delivery system (Orbit® SDS device) and prototypes in adult and juvenile canine eyes

对成年犬和幼犬眼中的Orbit®视网膜下给药系统(Orbit® SDS装置)及其原型进行评估

Kwok, Jennifer C; Gray, Alexa P; Sato, Yu; Savina, Svetlana; Dolgova, Natalia; Sudharsan, Raghavi; Boye, Sanford L; Boye, Shannon E; Reichenbacker, Sam; Meyer, Tom; Cummiskey, Kirsten Stoner; Beltran, William A

A Comprehensive Review of Clinically Applied Adeno-Associated Virus-Based Gene Therapies for Ocular Disease

临床应用腺相关病毒基因疗法治疗眼病的综合综述

Hinsch, Valerie G; Boye, Sanford L; Boye, Shannon E

Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS

经过基因工程改造的AAV衣壳转运突变体克服了老年中枢神经系统中的转导缺陷。

Ivette M Sandoval ,Christy M Kelley ,Luis Daniel Bernal-Conde ,Kathy Steece-Collier ,David J Marmion ,Marcus Davidsson ,Sean M Crosson ,Sanford L Boye ,Shannon E Boye ,Fredric P Manfredsson

Promotion of endoplasmic reticulum retrotranslocation by overexpression of E3 ubiquitin-protein ligase synoviolin 1 reduces endoplasmic reticulum stress and preserves cone photoreceptors in cyclic nucleotide-gated channel deficiency.

E3泛素蛋白连接酶synoviolin 1的过表达促进内质网逆向转位,从而降低内质网应激,并在环核苷酸门控通道缺陷中保护视锥细胞光感受器

Yang Fan, Ma Hongwei, Boye Sanford L, Boye Shannon E, Ding Xi-Qin

Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs

遗传性视网膜变性和非新生血管性年龄相关性黄斑变性:进展与未满足的需求

Duncan, Jacque L; Bowman, Angela; Laster, Amy; Gelfman, Claire; Birch, David G; Boye, Shannon E; Daiger, Stephen P; Del Priore, Lucian; Zack, Donald J; Handa, James T

Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion

在非人灵长类动物中,通过玻璃体内注射合理设计的AAV衣壳库,筛选出具有增强的视网膜转导能力和中和抗体逃避能力的变体。

Kellish, Patrick C; Marsic, Damien; Crosson, Sean M; Choudhury, Shreyasi; Scalabrino, Miranda L; Strang, Christianne E; Hill, Julie; McCullough, K Tyler; Peterson, James J; Fajardo, Diego; Gupte, Siddhant; Makal, Victoria; Kondratov, Oleksandr; Kondratova, Liudmyla; Iyer, Siva; Witherspoon, C Douglas; Gamlin, Paul D; Zolotukhin, Sergei; Boye, Sanford L; Boye, Shannon E