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

First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial

首次人体高剂量AAV9鞘内基因疗法治疗儿童CLN7疾病:一项1期、开放标签、单次递增剂量、非随机临床试验

Greenberg, Benjamin M; Minassian, Berge; Messahel, Souad; Edgar, Veronica Bordes; Lowden, Andrea; Dahshi, Hamza; Nettesheim, Emily R; Nguyen, Hoang H; Hughes, Samuel; Muthukumar, Alagar R; Srinivasan, Kalayarasan; Iannaccone, Susan; Varadarajan, Ganapathy; Gray, Steven J; Kayani, Saima N

Lafora disease gene therapy: EPM2A but not EPM2B overexpression results in Lafora body formation.

Lafora病基因治疗:EPM2A而非EPM2B过度表达会导致Lafora小体的形成。

Alao Esther O, Sheibani Mehrnaz, Wu Jun, Marriam Ummay, Evans Doretha, Kasiri Sahba, Verma Mayank, Nitschke Silvia, Nitschke Felix, Gray Steven J, Mitra Sharmistha, Minassian Berge A

Correction: Vyas et al. Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis. Int. J. Mol. Sci. 2023, 24, 14611

更正:Vyas 等人,《腺相关病毒 9 型介导的基因疗法治疗 AB 型 GM2 神经节苷脂病的疗效》,《国际分子科学杂志》,2023 年,24 卷,14611 页。

Vyas, Meera; Deschenes, Natalie M; Osmon, Karlaina J L; Chen, Zhilin; Ahmad, Imtiaz; Kot, Shalini; Thompson, Patrick; Richmond, Chris; Gray, Steven J; Walia, Jagdeep S

RNAi-Induced Expression of Paternal UBE3A

RNAi诱导父系UBE3A表达

Kang, Hye Ri; Zaric, Violeta; Rybalchenko, Volodymyr; Gray, Steven J; Butler, Ryan K

A Head-to-Head Comparison of AAV9 Biodistribution in Mice: Routes of Administration and Age Dependence.

AAV9 在小鼠体内的生物分布的直接比较:给药途径和年龄依赖性。

Rioux Matthew, Boitnott Andrea, Paduri Satvik, Hu Yuhui, Gray Steven J

Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II.

在新鉴定的猫粘脂病 II 型模型中,系统性 AAV 介导的基因治疗的年龄敏感性反应

Pyne Nettie K, Bagel Jessica, Shyng Charles, Odonnell Patricia, Miyadera Keiko, Srnak Jennifer, Swain Gary, Pesayco Jill P, Shelton G Diane, Assenmacher Charles-Antoine, Dickson Patricia, Stern Joshua A, Flanagan-Steet Heather, Gray Steven J, Bradbury Allison M

Improved AAV9-based gene therapy design for SURF1-related Leigh syndrome with minimal toxicity

针对SURF1相关Leigh综合征,改进的基于AAV9的基因治疗设计,毒性最小

Ling, Qinglan; Rioux, Matthew; Higgs, Harrison; Hu, Yuhui; Dwyer, Scarlett E; Gray, Steven J

Should patients with pre-existing anti-AAV antibodies be excluded from receiving intrathecally delivered AAV vectors?

对于体内已存在抗AAV抗体的患者,是否应将其排除在鞘内注射AAV载体之外?

Gray, Steven J

Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity.

聚焦超声可显著扩大 AAV 递送的 Cas9 的分布和活性

Gumusgoz Emrah, Kasiri Sahba, Youssef Ibrahim, Verma Mayank, Chopra Rajiv, Villarreal Acha Daniel, Wu Jun, Marriam Ummay, Alao Esther, Chen Xin, Guisso Dikran R, Gray Steven J, Shah Bhavya R, Minassian Berge A

Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy.

核黄素转运蛋白缺乏症:AAV9-SLC52A2 基因治疗作为一种新的治疗策略

Mei Cecilia, Magliocca Valentina, Chen Xin, Massey Keith, Gonzalez-Cordero Anai, Gray Steven J, Tartaglia Marco, Bertini Enrico Silvio, Corti Stefania, Compagnucci Claudia