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

Skeletal Muscle Biomarkers of Amyotrophic Lateral Sclerosis: A Large-Scale, Multi-Cohort Proteomic Study

肌萎缩侧索硬化症骨骼肌生物标志物:一项大规模多队列蛋白质组学研究

Dergai, Oleksandr; Wuu, Joanne; Koziczak-Holbro, Magdalena; Malaspina, Andrea; Granit, Volkan; Hernandez, Jessica P; Cooley, Anne; Sachdev, Ruchika; Yu, Lili; Bidinosti, Michael; Flotte, Ludivine; Nash, Mark; Jennings, Lori L; Berry, James D; Bruijn, Lucie I; Brachat, Sophie; Benatar, Michael

Immune Toxicities in AAV Gene Therapy: Overview for Clinicians

AAV基因治疗中的免疫毒性:临床医生概述

Parajuli, Shestruma; Gallagher, Thomas; Flotte, Terence R

Educational Case: Atypical presentation of primary hyperparathyroidism with brown tumor

教学案例:原发性甲状旁腺功能亢进症伴棕色瘤的非典型表现

Flotte, Ariel; Light, Jonathan; Klause, Harrison; Conran, Richard M

Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial

双载体rAAVrh8基因疗法治疗GM2神经节苷脂沉积症:一项1/2期试验

Eichler, Florian; Cataltepe, Oguz I; Daci, Rrita; Puri, Ajit S; Taghian, Toloo; Jiang, Xuntian; Shazeeb, Mohammed Salman; Kuhn, Anna; Hader, Asma; Celik, Hakki; Vardar, Zeynep; Lewis, Connor J; Artinian, Rebecca; Nagy, Amanda; Vachha, Behroze; Thompson, Robert; Gallagher, Thomas; Bateman, Scot; Parzych, Julia; Spanakis, Spiro G; Vaughn, Toby A; Pier, Kirsten; De Boever, Erika; Abbott, Mary-Alice; D Ambrosio, Eleonora; Kokoski, Danielle; Blackwood, Meghan; Drummond, Elise; Ratai, Eva-Maria; Townsend, Elise L; McLaughlin, Haley; Tifft, Cynthia J; Keeler, Allison M; Sena-Esteves, Miguel; Gray-Edwards, Heather L; Flotte, Terence R

Gene therapy then and now: A look back at changes in the field over the past 25 years

基因疗法的过去与现在:回顾过去25年该领域的变迁

Wang, Dan; Stevens, Gregg; Flotte, Terence R

Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy

一名接受高剂量rAAV9基因治疗后死于急性呼吸窘迫综合征的患者,其肺内皮细胞发生了转导。

Hordeaux, Juliette; Lamontagne, R Jason; Bandyopadhyay, Sushobhana; Bell, Peter; Wilson, James M; Flotte, Terence R

A compact base editor rescues AATD-associated liver and lung disease in mouse models

一种紧凑型碱基编辑器可挽救小鼠模型中与 AATD 相关的肝脏和肺部疾病

Jenny Gao; Bamidele, Nathan; Pires-Ferreira, Debora; Zheng, Chunwei; Destefano, April; Cheng, Haoyang; Tang, Qiushi; Cao, Yueying; Xie, Jun; Gao, Guangping; Gruntman, Alisha; Sontheimer, Erik; Flotte, Terence R; Xue, Wen

Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases

克服罕见病和超罕见病基因和细胞疗法商业化前期可行性障碍

Barrett, David; Cannon, Paula M; Mingozzi, Federico; Porteus, Matthew; Rivière, Isabelle; Flotte, Terence R

Neoadjuvant cobimetinib and atezolizumab with or without vemurafenib for stage III melanoma: outcomes and the impact of the microbiome from the NeoACTIVATE trial

新辅助治疗方案:cobimetinib 和 atezolizumab 联合或不联合 vemurafenib 治疗 III 期黑色素瘤:NeoACTIVATE 试验的结果及微生物组的影响

Block, Matthew S; Nelson, Garth D; Chen, Jun; Johnson, Stephen; Yang, Lu; Flotte, Thomas James; Grewal, Eric P; McWilliams, Robert R; Kottschade, Lisa A; Domingo-Musibay, Evidio; Markovic, Svetomir N; Dimou, Anastasios; Montane, Heather N; Piltin, Mara A; Price, Daniel L; Khariwala, Samir S; Hui, Jane Yuet Ching; Erskine, Courtney L; Strand, Carrie A; Zahrieh, David; Dong, Haidong; Hieken, Tina J

A single amino acid variant in the variable region I of AAV capsid confers liver detargeting.

AAV衣壳可变区I中的单个氨基酸变异可导致肝脏去靶向。

Xing Ruxiao, Xu Mengyao, Reil Darcy, Destefano April, Cui Mengtian, Liu Nan, Liang Jialing, Xu Guangchao, Luo Li, Xu Meiyu, Zhang Fang, Tai Phillip W L, Wei Yuquan, Gruntman Alisha M, Flotte Terence R, Gao Guangping, Wang Dan