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

Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression

来自非临床研究中AAV基因疗法治疗血友病的转化性见解:基因表达变异性和持久性的潜在机制

Fong, Sylvia; Swystun, Laura L; Batty, Paul; Lillicrap, David

Adeno-associated virus-based gene therapy for hemophilia-addressing the gaps

腺相关病毒介导的血友病基因治疗——弥补现有差距

Miesbach, Wolfgang; Batty, Paul; Chowdary, Pratima; Fong, Sylvia; Kaczmarek, Radoslaw; Leebeek, Frank W G; Long, Brian; Mahlangu, Johnny; Makris, Mike; Pierce, Glenn F; Pipe, Steven W; Srivastava, Alok; Voorberg, Jan; Peyvandi, Flora

Optimizing liver health before and after gene therapy for hemophilia A

血友病A基因治疗前后优化肝脏健康

Ragni, Margaret V; Mead, Henry; de Jong, Ype P; Kaczmarek, Radoslaw; Leavitt, Andrew D; Long, Brian; Nugent, Diane J; Sabatino, Denise E; Fong, Sylvia; von Drygalski, Annette; Walsh, Christopher E; Luxon, Bruce A

The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice

AAV5载体整合谱的纵向动力学及小鼠体内克隆扩增的评估

Ismail, Ashrafali Mohamed; Witt, Evan; Bouwman, Taren; Clark, Wyatt; Yates, Bridget; Franco, Matteo; Fong, Sylvia

Valoctocogene roxaparvovec gene transfer in participants with HIV

在 HIV 感染者中进行 Valoctocogene roxaparvovec 基因转移

Ragni, Margaret V; Majerus, Elaine; Fong, Sylvia; Yates, Bridget; Scheeler, Stephen; Razon, Lisa; Yu, Hua; Reddy, Divya B; Robinson, Tara M

Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action.

应用体外培养的原代肝细胞评估物种转化性和 AAV 转导作用机制

Liu Su, Razon Lisa, Ritchie Olivia, Sihn Choong-Ryoul, Handyside Britta, Berguig Geoffrey, Woloszynek Jill, Zhang Lening, Batty Paul, Lillicrap David, Agrawal Vishal, Cortesio Christa, Gebretsadik Kahsay, Akeefe Hassibullah, Colosi Peter, Kim Benjamin, Bunting Stuart, Fong Sylvia

Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter.

AAV5 BDD FVIII 构建体诱导内质网应激取决于肝脏特异性启动子的强度

Fong Sylvia, Handyside Britta, Sihn Choong-Ryoul, Liu Su, Zhang Lening, Xie Lin, Murphy Ryan, Galicia Nicole, Yates Bridget, Minto Wesley C, Vitelli Catherine, Harmon Danielle, Ru Yuanbin, Yu Guoying Karen, Escher Claudia, Vowinckel Jakob, Woloszynek Jill, Akeefe Hassib, Mahimkar Rajeev, Bullens Sherry, Bunting Stuart

Adenovirus-associated antibodies in UK cohort of hemophilia patients: A seroprevalence study of the presence of adenovirus-associated virus vector-serotypes AAV5 and AAV8 neutralizing activity and antibodies in patients with hemophilia A

英国血友病患者队列中腺病毒相关抗体:一项关于血友病A患者体内腺病毒相关病毒载体血清型AAV5和AAV8中和活性及抗体的血清流行病学研究

Stanford, Sophia; Pink, Ruth; Creagh, Desmond; Clark, Amanda; Lowe, Gillian; Curry, Nicola; Pasi, John; Perry, David; Fong, Sylvia; Hayes, Gregory; Chandrakumaran, Kandiah; Rangarajan, Savita

Skin Conduction Levels Differentiate Frontotemporal Dementia From Alzheimer's Disease

皮肤电导水平可区分额颞叶痴呆和阿尔茨海默病

Mendez, Mario F; Fong, Sylvia S; Ashla, Mark M; Jimenez, Elvira E; Carr, Andrew R

Effectiveness in hemophilia care through capacity building: the case of Yaoundé, Cameroon

通过能力建设提高血友病治疗效果:以喀麦隆雅温得为例

Lenting, Peter J; Fong, Sylvia; Willan, John; Agarwal, Gaurav; Varghese, Tara; Cairns, Karen; Baker, Peter; Curry, Nicola; Tran, Huyen; Deng, Lucy; Wood, Nicholas; Chen, Vivien M; Saslis, S; Dettoraki, A; Kapsimali, Z; Michalopoulou, A; Thymianou, S; Mazarakis, M; Pergantou, H; Mbanya, D; Tagny, C Tayou; Ndoumba, A; Moudourou, S