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

Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases

传递信息:转化mRNA疗法治疗肝脏遗传性代谢疾病

Gurung, Sonam; Perocheau, Dany; Ghosh, Roopkatha; Hart, Stephen L; Baruteau, Julien

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

Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice

巨噬细胞抑制剂氯膦酸盐可增强新生小鼠和幼年小鼠肝脏中慢病毒而非腺相关病毒载体或 mRNA 脂质纳米颗粒的转导

Loukia Touramanidou, Sonam Gurung, Claudiu A Cozmescu, Dany Perocheau, Dale Moulding, Patrick F Finn, Andrea Frassetto, Simon N Waddington, Paul Gissen, Julien Baruteau

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

Ex vivo precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases

离体精确切割的肝切片可模拟疾病表型并监测肝脏单基因疾病的治疗反应

Dany Perocheau, Sonam Gurung, Loukia Touramanidou, Claire Duff, Garima Sharma, Neil Sebire, Patrick F Finn, Alex Cavedon, Summar Siddiqui, Lisa Rice, Paolo G V Martini, Andrea Frassetto, Julien Baruteau

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors

根据预测腺相关病毒载体肝向性的能力对临床前肝脏模型进行评估

Adrian Westhaus, Marti Cabanes-Creus, Kimberley L Dilworth, Erhua Zhu, David Salas Gómez, Renina G Navarro, Anais K Amaya, Suzanne Scott, Magdalena Kwiatek, Alexandra L McCorkindale, Tara E Hayman, Silke Frahm, Dany P Perocheau, Bang Manh Tran, Elizabeth Vincan, Sharon L Wong, Shafagh A Waters, Geor

eDOL mHealth App and Web Platform for Self-monitoring and Medical Follow-up of Patients With Chronic Pain: Observational Feasibility Study

用于慢性疼痛患者自我监测和医疗随访的 eDOL 移动健康应用程序和网络平台:观察性可行性研究

Kerckhove, Nicolas; Delage, Noémie; Cambier, Sébastien; Cantagrel, Nathalie; Serra, Eric; Marcaillou, Fabienne; Maindet, Caroline; Picard, Pascale; Martiné, Gaelle; Deleens, Rodrigue; Trouvin, Anne-Priscille; Fourel, Lauriane; Espagne-Dubreuilh, Gaelle; Douay, Ludovic; Foulon, Stéphane; Dufraisse, Bénédicte; Gov, Christian; Viel, Eric; Jedryka, François; Pouplin, Sophie; Lestrade, Cécile; Combe, Emmanuel; Perrot, Serge; Perocheau, Dominique; De Brisson, Valentine; Vergne-Salle, Pascale; Mertens, Patrick; Pereira, Bruno; Djiberou Mahamadou, Abdoul Jalil; Antoine, Violaine; Corteval, Alice; Eschalier, Alain; Dualé, Christian; Attal, Nadine; Authier, Nicolas

Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism

基因疗法可恢复多巴胺转运蛋白的表达并改善婴儿帕金森病 iPSC 和小鼠模型中的病理

Joanne Ng, Serena Barral, Carmen De La Fuente Barrigon, Gabriele Lignani, Fatma A Erdem, Rebecca Wallings, Riccardo Privolizzi, Giada Rossignoli, Haya Alrashidi, Sonja Heasman, Esther Meyer, Adeline Ngoh, Simon Pope, Rajvinder Karda, Dany Perocheau, Julien Baruteau, Natalie Suff, Juan Antinao Diaz, 

Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders

Beclin-1介导的自噬激活改善近端和远端尿素循环障碍

Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta, Michael Orford, Youssef Khalil, Simon Eaton, Philippa B Mills, Simon N Waddington, Carmine Settembre, Andrés F Muro, Julien Baruteau, Nicola

The exosome journey: from biogenesis to uptake and intracellular signalling

外泌体的旅程:从生物发生到摄取和细胞内信号传导

Gurung, Sonam; Perocheau, Dany; Touramanidou, Loukia; Baruteau, Julien