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

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria

mRNA疗法可纠正临床前精氨酸琥珀酸尿症中谷胱甘肽代谢缺陷并恢复尿素生成。

Gurung, Sonam; Timmermand, Oskar Vilhelmsson; Perocheau, Dany; Gil-Martinez, Ana Luisa; Minnion, Magdalena; Touramanidou, Loukia; Fang, Sherry; Messina, Martina; Khalil, Youssef; Spiewak, Justyna; Barber, Abigail R; Edwards, Richard S; Pinto, Patricia Lipari; Finn, Patrick F; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G V; Ridout, Deborah; Heywood, Wendy; Hargreaves, Ian; Heales, Simon; Mills, Philippa B; Waddington, Simon N; Gissen, Paul; Eaton, Simon; Ryten, Mina; Feelisch, Martin; Frassetto, Andrea; Witney, Timothy H; Baruteau, Julien

The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria

运动障碍的发生率随年龄增长而增加,这与精氨酸琥珀酸尿症中轻微且有限的神经影像学异常形成对比。

Gurung, Sonam; Karamched, Saketh; Perocheau, Dany; Seunarine, Kiran K; Baldwin, Tom; Alrashidi, Haya; Touramanidou, Loukia; Duff, Claire; Elkhateeb, Nour; Stepien, Karolina M; Sharma, Reena; Morris, Andrew; Hartley, Thomas; Crowther, Laura; Grunewald, Stephanie; Cleary, Maureen; Mundy, Helen; Chakrapani, Anupam; Batzios, Spyros; Davison, James; Footitt, Emma; Tuschl, Karin; Lachmann, Robin; Murphy, Elaine; Santra, Saikat; Uudelepp, Mari-Liis; Yeo, Mildrid; Finn, Patrick F; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G V; Frassetto, Andrea; Heales, Simon; Mills, Philippa B; Gissen, Paul; Clayden, Jonathan D; Clark, Christopher A; Eaton, Simon; Kalber, Tammy L; Baruteau, Julien

Biomarkers of haemodynamic severity of systemic sclerosis-associated pulmonary arterial hypertension by serum proteome analysis

通过血清蛋白质组分析检测系统性硬化症相关肺动脉高压血流动力学严重程度的生物标志物

Sanges, Sébastien; Rice, Lisa; Tu, Ly; Valenzi, Eleanor; Cracowski, Jean-Luc; Montani, David; Mantero, Julio C; Ternynck, Camille; Marot, Guillemette; Bujor, Andreea M; Hachulla, Eric; Launay, David; Humbert, Marc; Guignabert, Christophe; Lafyatis, Robert

Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model

合成mRNA可挽救患者成纤维细胞和鼠模型中的极长链酰基辅酶A脱氢酶缺乏症。

Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Aliu, Ermal; Shi, Huifang; Basu, Shakuntala; Kochersperger, Catherine; Van't Land, Clinton; Karunanidhi, Anuradha; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry

Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine model

信使RNA可挽救患者和鼠模型成纤维细胞中的中链酰基辅酶A脱氢酶缺乏症

Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Basu, Shakuntala; Aliu, Ermal; Shi, Huifang; Kochersberger, Catherine; Karunanidhi, Anuradha; Van't Land, Clinton; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; DeAntonis, Christine; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry

Eukaryotic initiation factor 6 regulates mechanical responses in endothelial cells

真核起始因子 6 调节内皮细胞的机械反应

Adam N Keen, Luke A Payne #, Vedanta Mehta #, Alistair Rice #, Lisa J Simpson, Kar Lai Pang, Armando Del Rio Hernandez, John S Reader, Ellie Tzima

Correction: Serum biomarker for diagnostic evaluation of pulmonary arterial hypertension in systemic sclerosis

更正:用于系统性硬化症肺动脉高压诊断评估的血清生物标志物

Rice, Lisa M; Mantero, Julio C; Stratton, Eric A; Warburton, Rod; Roberts, Kari; Hill, Nicholas; Simms, Robert W; Domsic, Robyn; Farber, Harrison W; Lafyatis, Robert

Mitophagy-dependent macrophage reprogramming protects against kidney fibrosis

线粒体自噬依赖性巨噬细胞重编程可预防肾纤维化

Divya Bhatia ,Kuei-Pin Chung ,Kiichi Nakahira ,Edwin Patino ,Michelle C Rice ,Lisa K Torres ,Thangamani Muthukumar ,Augustine Mk Choi ,Oleh M Akchurin ,Mary E Choi

Reduction of the expression of the late-onset Alzheimer's disease (AD) risk-factor BIN1 does not affect amyloid pathology in an AD mouse model

降低晚发性阿尔茨海默病 (AD) 风险因子 BIN1 的表达不会影响 AD 小鼠模型中的淀粉样蛋白病理

Robert J Andrew, Pierre De Rossi, Phuong Nguyen, Haley R Kowalski, Aleksandra J Recupero, Thomas Guerbette, Sofia V Krause, Richard C Rice, Lisa Laury-Kleintop, Steven L Wagner, Gopal Thinakaran

Skin Gene Expression Is Prognostic for the Trajectory of Skin Disease in Patients With Diffuse Cutaneous Systemic Sclerosis

皮肤基因表达可预测弥漫性皮肤系统性硬化症患者的皮肤疾病发展轨迹

Stifano, Giuseppina; Sornasse, Thierry; Rice, Lisa M; Na, Leo; Chen-Harris, Haiyin; Khanna, Dinesh; Jahreis, Angelika; Zhang, Yuqing; Siegel, Jeff; Lafyatis, Robert