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

AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia

AAV2介导的玻璃体内递送外显子特异性U1 snRNA可挽救家族性自主神经功能障碍患者的视神经病变

Chekuri, Anil; Kondabolu, Krishnakanth; Kirchner, Emily G; Koli, Swanand; Chagnon, Matthew; Krasniqi-Vanmeter, Drenushe; Stern, Max E; Bolduc, Jessica; Romano, Giulia; Pagani, Franco; Vandenberghe, Luk H; Morini, Elisabetta; Slaugenhaupt, Susan A

Computed tomography and [(18)F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates

计算机断层扫描和[(18)F]-FDG PET成像为非人灵长类动物COVID-19发病机制和治疗评估提供了额外的读数。

Naninck, Thibaut; Kahlaoui, Nidhal; Lemaitre, Julien; Maisonnasse, Pauline; De Mori, Antoine; Pascal, Quentin; Contreras, Vanessa; Marlin, Romain; Relouzat, Francis; Delache, Benoît; Hérate, Cécile; Aldon, Yoann; van Gils, Marit; Zabaleta, Nerea; Ho Tsong Fang, Raphaël; Bosquet, Nathalie; Sanders, Rogier W; Vandenberghe, Luk H; Chapon, Catherine; Le Grand, Roger

Selective retinal ganglion cell loss and optic neuropathy in a humanized mouse model of familial dysautonomia

在家族性自主神经功能障碍的人源化小鼠模型中,选择性视网膜神经节细胞丢失和视神经病变

Chekuri, Anil; Logan, Emily M; Krauson, Aram J; Salani, Monica; Ackerman, Sophie; Kirchner, Emily G; Bolduc, Jessica M; Wang, Xia; Dietrich, Paula; Dragatsis, Ioannis; Vandenberghe, Luk H; Slaugenhaupt, Susan A; Morini, Elisabetta

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

在甲基丙二酸血症小鼠模型中,ImmTOR纳米颗粒在首次和重复给药后可增强AAV转基因的表达。

Ilyinskii, Petr O; Michaud, Alicia M; Rizzo, Gina L; Roy, Christopher J; Leung, Sheldon S; Elkins, Stephanie L; Capela, Teresa; Chowdhury, Aparajita; Li, Lina; Chandler, Randy J; Manoli, Irini; Andres-Mateos, Eva; Johnston, Lloyd P M; Vandenberghe, Luk H; Venditti, Charles P; Kishimoto, Takashi Kei

Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration

基因疗法在NMNAT1相关视网膜变性小鼠模型中保护了视网膜的结构和功能。

Greenwald, Scott H; Brown, Emily E; Scandura, Michael J; Hennessey, Erin; Farmer, Raymond; Pawlyk, Basil S; Xiao, Ru; Vandenberghe, Luk H; Pierce, Eric A

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

治疗性腺相关病毒(AAV)基因转移至神经系统:临床现实

Hudry, Eloise; Vandenberghe, Luk H

AAV Engineering Identifies a Species Barrier That Highlights a Portal to the Brain

AAV工程发现了一种物种屏障,该屏障揭示了通往大脑的入口。

Vandenberghe, Luk H

Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy

腺相关病毒衣壳的填充:利用冷冻电镜评估填充成功率

Subramanian, Suriyasri; Maurer, Anna C; Bator, Carol M; Makhov, Alexander M; Conway, James F; Turner, Kevin B; Marden, James H; Vandenberghe, Luk H; Hafenstein, Susan L

Quantitative and Digital Droplet-Based AAV Genome Titration

定量和数字化液滴基AAV基因组滴定

Sanmiguel, Julio; Gao, Guangping; Vandenberghe, Luk H

Breaking and Sealing Barriers in Retinal Gene Therapy

视网膜基因治疗中的障碍突破与修复

Miller, Joan W; Vandenberghe, Luk H