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

New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment

拉福拉病的新治疗策略:静脉注射 VAL-1221 治疗的安全性、有效性、药代动力学和代谢组学特征评估

Muccioli, Lorenzo; Tappatà, Maria; Farolfi, Andrea; Singh, Pankaj K; Pasini, Elena; Mazzone, Serena; Esposito, Erika; Motta, Lorenzo; D'Agati, Olivia M; Angeloni, Sofia; Caravelli, Alice; Cancellerini, Chiara; Minardi, Raffaella; Pizzi, Eleonora; Carelli, Valerio; Tonon, Caterina; Fiori, Jessica; Gentry, Matthew S; Vignatelli, Luca; Michelucci, Roberto; Bisulli, Francesca

Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI

利用饱和转移磁共振成像技术绘制庞贝病中糖原积累及治疗效果图

Zeng, Qing; Li, Yuguo; Timm, Derek; Johnson, Tyler; Mehta, Nickita; Martin, Lukas; Fox, Brian; van Zijl, Peter C M; Walter, Glenn A; Rosenberg, Jens T; Kooi, Craig W Vander; Corti, Manuela; Gentry, Matthew S; Sun, Ramon C; Byrne, Barry J; Yadav, Nirbhay N

The biochemical dynamics of the glycogen phosphatase laforin directly impact brain metabolism

糖原磷酸酶拉福林的生化动力学直接影响脑代谢

Brewer, M Kathryn; Donohue, Katherine J; Singh, Pankaj K; Raththagala, Madushi; Simmons, Zoe R; Wayne, Jeremiah L; Li, Sheng; Viana, Rosa; Segvich, Dyann M; Contreras, Christopher J; Cantrell, Alex R; Sanz, Pascual; Sun, Ramon C; Vander Kooi, Craig W; Roach, Peter J; DePaoli-Roach, Anna; Gentry, Matthew S

Spatial mapping of the brain metabolome lipidome and glycome.

脑代谢组、脂质组和糖组的空间映射

Clarke Harrison A, Ma Xin, Shedlock Cameron J, Medina Terrymar, Hawkinson Tara R, Wu Lei, Ribas Roberto A, Keohane Shannon, Ravi Sakthivel, Bizon Jennifer L, Burke Sara N, Abisambra Jose Francisco, Merritt Matthew E, Prentice Boone M, Vander Kooi Craig W, Gentry Matthew S, Chen Li, Sun Ramon C

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.

新生儿全身基因疗法可恢复庞贝病大鼠模型的心肺功能。

Fuller David D, Rana Sabhya, Thakre Prajwal P, Benevides Ethan S, Pope Megan K, Todd Adrian G, Jensen Victoria N, Vaught Lauren, Cloutier Denise A, Ribas Roberto A, Larson Reece C, Gentry Matthew S, Sun Ramon C, Chandran Vijay, Corti Manuela, Falk Darin J, Byrne Barry J

Neuromodulatory control of energy reserves in dopaminergic neurons

多巴胺能神经元能量储备的神经调节控制

Pulido, Camila; Gentry, Matthew S; Ryan, Timothy A

GDP-mannose 4,6-dehydratase is a key driver of MYCN-amplified neuroblastoma core fucosylation and tumorigenesis

GDP-甘露糖4,6-脱水酶是MYCN扩增的神经母细胞瘤核心岩藻糖基化和肿瘤发生的关键驱动因素

Zhu, Beibei; Pitts, Michelle G; Buoncristiani, Michael D; Bryant, Lindsay T; Lopez-Nunez, Oscar; Gurria, Juan P; Shedlock, Cameron; Ribas, Roberto; Keohane, Shannon; Liu, Jinpeng; Wang, Chi; Gentry, Matthew S; Shelman, Nathan R; Allison, Derek B; Evers, B Mark; Sun, Ramon C; Rellinger, Eric J

Discovery and Evaluation of Active Site-Directed, Potent, and Selective Sulfophenyl Acetic Amide-Based Inhibitors for the Laforin Phosphatase

发现和评价活性位点导向的、高效、选择性磺基苯乙酸酰胺类拉福林磷酸酶抑制剂

Lin, Jianping; He, Rongjun; Qu, Zihan; Dong, Jiajun; Krabill, Aaron D; Wu, Li; Bai, Yunpeng; Conroy, Lindsey R; Bruntz, Ronald C; Miao, Yiming; Jassim, Brenson A; Babalola, Benjamin; Nguele Meke, Frederick Georges Bernard; Sun, Ramon; Gentry, Matthew S; Zhang, Zhong-Yin

Predictive modeling of ARDS mortality integrating biomarker/cytokine, clinical and metabolomic data

整合生物标志物/细胞因子、临床和代谢组学数据的ARDS死亡率预测模型

Rafikov, Ruslan; Thompson, Debrah M; Rafikova, Olga; Camp, Sara M; Ribas, Roberto A; Sun, Ramon C; Gentry, Matthew S; Casanova, Nancy G; Garcia, Joe G N

Dynamics of amylopectin granule accumulation during the course of chronic Toxoplasma infection is linked to intra-cyst bradyzoite replication

慢性弓形虫感染过程中支链淀粉颗粒的积累动态与囊内缓殖子复制有关。

Tripathi, Aashutosh; Donkin, Ryan W; Miracle, Joy S; Murphy, Robert D; Gentry, Matthew S; Patwardhan, Abhijit; Sinai, Anthony P