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

Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec

一名接受onasemnogene abeparvovec治疗的脊髓性肌萎缩症患儿出现毛细胞型星形细胞瘤。

Holzwarth, Dorothea; Calaminus, Gabriele; Friese, Johannes; Sejersen, Thomas; Büning, Hildegard; John-Neek, Philipp; Bastone, Antonella Lucía; Rothe, Michael; Mansfield, Keith; Libertini, Silvana; Dubost, Valérie; Kuzmiski, Brent; Alecu, Iulian; Labik, Ivan; Kirschner, Janbernd

Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: Insights into organ toxicities

鞘内注射9型腺相关病毒载体后非人灵长类动物组织转录组的变化:对器官毒性的深入了解

Aihara, Fumiaki; Mardo, Matthew; Ruda, Vera; Rio, Tony Del; Bigot, Karine; Singer, Jochen; Onishi-Seebacher, Megumi; Couttet, Philippe; Mansfield, Keith; Hudry, Eloise

Impact of pre-existing immunity on safety and biodistribution of a single AAV9 vectorintrathecal injection in cynomolgus monkeys.

既往免疫对食蟹猴单次鞘内注射 AAV9 载体的安全性和生物分布的影响。

Vono Maria, Rio Tony Del, Magdaleno Eduardo, Loll Nathalie R, Jarvis Philip, Walter Catherine, Kikkawa Rie, Mansfield Keith, McBlane Fraser, Sirivelu Madhu P, Ozsolak Fatih, Brees Dominique, Hudry Eloise, Rubic-Schneider Tina, Tukov Francis Fonyuy, Huang Guanrong

Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec

一名患有脊髓性肌萎缩症的儿童,接受了onasemnogene abeparvovec治疗,结果发现其脊髓出现上皮样肿瘤。

Retson, Laura; Tiwari, Nishant; Vaughn, Jennifer; Bernes, Saunder; Adelson, P David; Mansfield, Keith; Libertini, Silvana; Kuzmiski, Brent; Alecu, Iulian; Gabriel, Richard; Mangum, Ross

Two cases of severe pulmonary toxicity from highly active mesothelin-directed CAR T cells

两例由高活性间皮素靶向CAR-T细胞引起的严重肺毒性病例

Haas, Andrew R; Golden, Ryan J; Litzky, Leslie A; Engels, Boris; Zhao, Linlin; Xu, Fangmin; Taraszka, John A; Ramones, Melissa; Granda, Brian; Chang, Wan-Jung; Jadlowsky, Julie; Shea, Kim-Marie; Runkle, Adam; Chew, Anne; Dowd, Emily; Gonzalez, Vanessa; Chen, Fang; Liu, Xiaojun; Fang, Chongyun; Jiang, Shuguang; Davis, Megan M; Sheppard, Neil C; Zhao, Yangbing; Fraietta, Joseph A; Lacey, Simon F; Plesa, Gabriela; Melenhorst, J Joseph; Mansfield, Keith; Brogdon, Jennifer L; Young, Regina M; Albelda, Steven M; June, Carl H; Tanyi, Janos L

Liver injury in cynomolgus monkeys following intravenous and intrathecal scAAV9 gene therapy delivery.

食蟹猴经静脉和鞘内注射 scAAV9 基因治疗后出现肝损伤

Hudry Eloise, Aihara Fumiaki, Meseck Emily, Mansfield Keith, McElroy Cameron, Chand Deepa, Tukov Francis Fonyuy, Penraat Kelley

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys

单剂量鞘内注射Onasemnogene Abeparvovec对食蟹猴背根神经节的毒性

Tukov, Francis Fonyuy; Mansfield, Keith; Milton, Mark; Meseck, Emily; Penraat, Kelley; Chand, Deepa; Hartmann, Andreas

Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques

鞘内注射sc-AAV9-CB-GFP:食蟹猴单次给药后以全身分布为主

Meseck, Emily K; Guibinga, Ghiabe; Wang, Stephen; McElroy, Cameron; Hudry, Eloise; Mansfield, Keith

A single dose of peripherally infused EGFRvIII-directed CAR T cells mediates antigen loss and induces adaptive resistance in patients with recurrent glioblastoma

单次外周输注EGFRvIII靶向CAR-T细胞可导致复发性胶质母细胞瘤患者出现抗原丢失并诱导适应性耐药。

O'Rourke, Donald M; Nasrallah, MacLean P; Desai, Arati; Melenhorst, Jan J; Mansfield, Keith; Morrissette, Jennifer J D; Martinez-Lage, Maria; Brem, Steven; Maloney, Eileen; Shen, Angela; Isaacs, Randi; Mohan, Suyash; Plesa, Gabriela; Lacey, Simon F; Navenot, Jean-Marc; Zheng, Zhaohui; Levine, Bruce L; Okada, Hideho; June, Carl H; Brogdon, Jennifer L; Maus, Marcela V

Feasibility of intravitreal injections and ophthalmic safety assessment in marmoset (Callithrix jacchus) monkeys

狨猴(Callithrix jacchus)玻璃体内注射的可行性及眼科安全性评估

Korbmacher, Birgit; Atorf, Jenny; Fridrichs-Gromoll, Stephanie; Hill, Marilyn; Korte, Sven; Kremers, Jan; Mansfield, Keith; Mecklenburg, Lars; Pilling, Andrew; Wiederhold, Andreas