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

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.

FLT201 是一种新型的肝脏靶向 AAV 基因治疗候选药物,用于治疗 1 型戈谢病。

Comper Fabrizio, Miranda Carlos J, Liou Benjamin, Dodev Tihomir, Jeyakumar Jey M, Canavese Miriam, Cocita Clement, Khoshrou Khashayar, Tiscornia Gustavo, Chisari Elisa, Stotter Emmaline, Shehu Erald, Sridharan Sudharsan, Yu I-Mei, Pandya Jalpa, Khinder Jaminder, Northcott Natalie, Kalcheva Petya, Correia Samantha, Sun Ying, Dane Allison P, Sheridan Rose, Nathwani Amit C, Corbau Romuald

Patient-Specific Midbrain Organoids with CRISPR Correction Reveal Disease Mechanisms and Enable Therapeutic Evaluation in Neuronopathic Gaucher Disease

利用 CRISPR 技术进行基因编辑的患者特异性中脑类器官揭示了神经病变型戈谢病的疾病机制,并可用于治疗评估。

Lin, Yi; Liou, Benjamin; Fannin, Venette; Adler, Stuart; Mayhew, Christopher N; Hammonds, Jason E; Hu, Yueh-Chiang; Tchieu, Jason; Zhang, Wujuan; Zhao, Xueheng; Beres, Rebecca L; Setchell, Kenneth Dr; Kaynak, Ahmet; Qi, Xiaoyang; Feldman, Ricardo A; Sun, Ying

PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases

PGRN 缺乏会加剧 GBA1 突变相关的病理和疾病,而一种可穿透血脑屏障的 PGRN 衍生物则具有保护作用。

Zhao, Xiangli; Lin, Yi; Liou, Benjamin; Fu, Wenyu; Jian, Jinlong; Fannie, Venette; Zhang, Wujuan; Setchell, Kenneth D R; Grabowski, Gregory A; Sun, Ying; Liu, Chuan-Ju

Combination of acid β-glucosidase mutation and Saposin C deficiency in mice reveals Gba1 mutation dependent and tissue-specific disease phenotype

小鼠酸性β-葡萄糖苷酶突变和Saposin C缺乏的组合揭示了Gba1突变依赖性和组织特异性疾病表型

Liou, Benjamin; Zhang, Wujuan; Fannin, Venette; Quinn, Brian; Ran, Huimin; Xu, Kui; Setchell, Kenneth D R; Witte, David; Grabowski, Gregory A; Sun, Ying

The effect of manganese exposure in Atp13a2-deficient mice.

锰暴露对Atp13a2缺陷小鼠的影响

Fleming Sheila M, Santiago Nicholas A, Mullin Elizabeth J, Pamphile Shanta, Karkare Swagata, Lemkuhl Andrew, Ekhator Osunde R, Linn Stephen C, Holden John G, Aga Diana S, Roth Jerome A, Liou Benjamin, Sun Ying, Shull Gary E, Schultheis Patrick J

Modulating ryanodine receptors with dantrolene attenuates neuronopathic phenotype in Gaucher disease mice

用丹曲林调节兰尼碱受体可减轻戈谢病小鼠的神经病变表型

Liou, Benjamin; Peng, Yanyan; Li, Ronghua; Inskeep, Venette; Zhang, Wujuan; Quinn, Brian; Dasgupta, Nupur; Blackwood, Rachel; Setchell, Kenneth D R; Fleming, Sheila; Grabowski, Gregory A; Marshall, John; Sun, Ying

Progression of Behavioral and CNS Deficits in a Viable Murine Model of Chronic Neuronopathic Gaucher Disease

慢性神经病变戈谢病小鼠模型中行为和中枢神经系统缺陷的进展

Dai, Mei; Liou, Benjamin; Swope, Brittany; Wang, Xiaohong; Zhang, Wujuan; Inskeep, Venette; Grabowski, Gregory A; Sun, Ying; Pan, Dao

Participation of asparagine 370 and glutamine 235 in the catalysis by acid beta-glucosidase: the enzyme deficient in Gaucher disease

天冬酰胺370和谷氨酰胺235参与酸性β-葡萄糖苷酶的催化作用:该酶在戈谢病中缺乏

Liou, Benjamin; Grabowski, Gregory A