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

From Radiocopper to Cold Copper: Mechanistic Modeling and Simulation to Define Clinical Response Criteria and Biomarkers for VTX-801 in Wilson Disease

从放射性铜到冷铜:利用机制建模和模拟确定VTX-801治疗威尔逊病的临床反应标准和生物标志物

Lindauer, Andreas; Benichou, Bernard; González Aseguinolaza, Gloria; Combal, Jean-Philippe

Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy

38例PFIC3患者的预后:基因型和熊去氧胆酸治疗反应的影响

Gonzales, Emmanuel; Gardin, Antoine; Almes, Marion; Darmellah-Remil, Amaria; Seguin, Hanh; Mussini, Charlotte; Franchi-Abella, Stéphanie; Duché, Mathieu; Ackermann, Oanez; Thébaut, Alice; Habes, Dalila; Hermeziu, Bogdan; Lapalus, Martine; Falguières, Thomas; Combal, Jean-Philippe; Benichou, Bernard; Valero, Sonia; Davit-Spraul, Anne; Jacquemin, Emmanuel

Rescue of infant progressive familial intrahepatic cholestasis type 3 mice by repeated dosing of AAV gene therapy

通过重复给予AAV基因疗法挽救患有进行性家族性肝内胆汁淤积症3型的幼年小鼠。

Weber, Nicholas D; Odriozola, Leticia; Ros-Gañán, Irene; García-Porrero, Guillermo; Salas, David; Argemi, Josepmaria; Combal, Jean-Philippe; Kishimoto, Takashi K; González-Aseguinolaza, Gloria

High value of (64)Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease

高 (64)Cu 值可作为评估威尔逊病基因治疗后生理性铜排泄恢复情况的工具

Murillo, Oihana; Collantes, Maria; Gazquez, Cristina; Moreno, Daniel; Hernandez-Alcoceba, Ruben; Barberia, Miren; Ecay, Margarita; Tamarit, Blanche; Douar, Anne; Ferrer, Veronica; Combal, Jean Philippe; Peñuelas, Ivan; Bénichou, Bernard; Gonzalez-Aseguinolaza, Gloria

Optimising the IgG-degrading enzyme treatment regimen for enhanced adeno-associated virus transduction in the presence of neutralising antibodies

优化 IgG 降解酶治疗方案以在存在中和抗体的情况下增强腺相关病毒的转导

Irene Ros-Gañán, Mirja Hommel, Laia Trigueros-Motos, Blanche Tamarit, Estefanía Rodríguez-García, David Salas, Guiomar Pérez, Anne Douar, Jean Philippe Combal, Bernard Benichou, Veronica Ferrer, Gloria González-Aseguinolaza

Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice

基因治疗与氮清除剂预处理相结合可纠正婴儿瓜氨酸血症 1 型小鼠的生化和行为异常

Andrea Bazo, Aquilino Lantero, Itsaso Mauleón, Leire Neri, Martin Poms, Johannes Häberle, Ana Ricobaraza, Bernard Bénichou, Jean-Philippe Combal, Gloria Gonzalez-Aseguinolaza, Rafael Aldabe

Preliminary Results of the "Capasquelet" Technique for Managing Femoral Bone Defects-Combining a Masquelet Induced Membrane and Capanna Vascularized Fibula with an Allograft

“Capasquelet”技术治疗股骨骨缺损的初步结果——结合Masquelet诱导膜和Capanna血管化腓骨以及同种异体移植

Combal, Alexis; Thuau, François; Fouasson-Chailloux, Alban; Arrigoni, Pierre-Paul; Baud'huin, Marc; Duteille, Franck; Crenn, Vincent

Could enteral nutrition improve the outcome of patients with haematological malignancies undergoing allogeneic haematopoietic stem cell transplantation? A study protocol for a randomized controlled trial (the NEPHA study)

肠内营养能否改善接受异基因造血干细胞移植的血液系统恶性肿瘤患者的预后?一项随机对照试验的研究方案(NEPHA 研究)

Lemal, Richard; Cabrespine, Aurélie; Pereira, Bruno; Combal, Cécile; Ravinet, Aurélie; Hermet, Eric; Bay, Jacques-Olivier; Bouteloup, Corinne