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

Inhaled DNAI1 mRNA therapy for treatment of primary ciliary dyskinesia.

吸入式 DNAI1 mRNA 疗法治疗原发性纤毛运动障碍

Hennig Mirko, Bhattacharjee Rumpa B, Agarwal Ishita, Alfaifi Ali, Casillas Jade E, Chavez Sofia, Ishimaru Daniella, Liston David, Mohapatra Sakya, Molla Touhidul, Pathare Suyog, Sidhu Maninder S, Wang Peng, Wang Zechen, Lombana T Noelle, Kharitonov Vladimir G, Couch Jessica A, Lockhart David J, Wustman Brandon A

Lipid nanoparticle-encapsulated Dnai1 mRNA rescues ciliary activity in primary ciliary dyskinesia mouse cell models

脂质纳米颗粒包裹的Dnai1 mRNA可挽救原发性纤毛运动障碍小鼠细胞模型中的纤毛活动。

Smith, Amanda J; Sears, Patrick R; Hennig, Mirko; Bhattacharjee, Rumpa B; Yin, Weining; Golliher, Hannah; Ishimaru, Daniella; Lombana, T Noelle; Lockhart, David J; Wustman, Brandon A; Ostrowski, Lawrence E

Treatment-Free Remission Outcomes in a BCR::ABL1 Digital PCR Selected Clinical Cohort of CML Patients

在 BCR::ABL1 数字 PCR 选择的 CML 患者临床队列中,无治疗缓解的结果

Kockerols, C; Valk, P J M; Hogenbirk, P; Geelen, I; Blijlevens, N M A; Janssen, J J W M; Hoogendoorn, M; Kersting, S; Klein, S K; Daenen, L G M; Donker, M; Te Boekhorst, P A W; Jie, K-S G; Corsten, M; Cruijsen, M J; Levenga, H; Smit, W M; Levin, M-D; de Jongh, E; de Jonge, S; Vlot, A J; Durian, M F; Zwaginga, J J; Mohlmann, M; Wustman, T J; Blommers, R; Cornelissen, J J; Westerweel, P E

In vivo editing of lung stem cells for durable gene correction in mice

利用小鼠肺干细胞进行体内编辑以实现持久基因校正

Sun, Yehui; Chatterjee, Sumanta; Lian, Xizhen; Traylor, Zachary; Sattiraju, Sandhya R; Xiao, Yufen; Dilliard, Sean A; Sung, Yun-Chieh; Kim, Minjeong; Lee, Sang M; Moore, Stephen; Wang, Xu; Zhang, Di; Wu, Shiying; Basak, Pratima; Wang, Jialu; Liu, Jing; Mann, Rachel J; LePage, David F; Jiang, Weihong; Abid, Shadaan; Hennig, Mirko; Martinez, Anna; Wustman, Brandon A; Lockhart, David J; Jain, Raksha; Conlon, Ronald A; Drumm, Mitchell L; Hodges, Craig A; Siegwart, Daniel J

Evidence that small molecule enhancement of β-hexosaminidase activity corrects the behavioral phenotype in Dutch APP(E693Q) mice through reduction of ganglioside-bound Aβ

证据表明,小分子增强β-己糖胺酶活性可通过减少神经节苷脂结合的Aβ来纠正荷兰APP(E693Q)小鼠的行为表型。

Knight, E M; Williams, H N; Stevens, A C; Kim, S H; Kottwitz, J C; Morant, A D; Steele, J W; Klein, W L; Yanagisawa, K; Boyd, R E; Lockhart, D J; Sjoberg, E R; Ehrlich, M E; Wustman, B A; Gandy, S

The Origin and Evolution of the Plant Cell Surface: Algal Integrin-Associated Proteins and a New Family of Integrin-Like Cytoskeleton-ECM Linker Proteins

植物细胞表面的起源与演化:藻类整合素相关蛋白和一类新的整合素样细胞骨架-细胞外基质连接蛋白

Becker, Burkhard; Doan, Jean Michel; Wustman, Brandon; Carpenter, Eric J; Chen, Li; Zhang, Yong; Wong, Gane K-S; Melkonian, Michael

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

当与输注阿加糖酶联合使用时,口服米格司他盐酸盐可提高法布里病患者体内活性α-半乳糖苷酶A的全身暴露量和组织水平。

Warnock, David G; Bichet, Daniel G; Holida, Myrl; Goker-Alpan, Ozlem; Nicholls, Kathy; Thomas, Mark; Eyskens, Francois; Shankar, Suma; Adera, Mathews; Sitaraman, Sheela; Khanna, Richie; Flanagan, John J; Wustman, Brandon A; Barth, Jay; Barlow, Carrolee; Valenzano, Kenneth J; Lockhart, David J; Boudes, Pol; Johnson, Franklin K

Bringing dead proteins back to life

让死去的蛋白质复活

A Wustman, Brandon; Steele, John W; Sjoberg, Eric R; Stevens, Anthony C

A GCase chaperone improves motor function in a mouse model of synucleinopathy

GCase 分子伴侣可改善突触核蛋白病小鼠模型的运动功能

Franziska Richter, Sheila M Fleming, Melanie Watson, Vincent Lemesre, Lee Pellegrino, Brian Ranes, Chunni Zhu, Farzad Mortazavi, Caitlin K Mulligan, Pedrom C Sioshansi, Sindalana Hean, Krystal De La Rosa, Richie Khanna, John Flanagan, David J Lockhart, Brandon A Wustman, Sean W Clark, Marie-François

The pharmacological chaperone AT2220 increases the specific activity and lysosomal delivery of mutant acid alpha-glucosidase, and promotes glycogen reduction in a transgenic mouse model of Pompe disease

药理学伴侣 AT2220 可增加突变酸性 α-葡萄糖苷酶的特异性活性和溶酶体递送,并促进庞贝病转基因小鼠模型中的糖原减少

Richie Khanna, Allan C Powe Jr, Yi Lun, Rebecca Soska, Jessie Feng, Rohini Dhulipala, Michelle Frascella, Anadina Garcia, Lee J Pellegrino, Su Xu, Nastry Brignol, Matthew J Toth, Hung V Do, David J Lockhart, Brandon A Wustman, Kenneth J Valenzano