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

Preclinical development of TAK-754, a high-performance AAV8-based vector expressing coagulation factor VIII

TAK-754 的临床前开发，TAK-754 是一种基于 AAV8 的高性能载体，可表达凝血因子 VIII

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101424
Lengler, Johannes; Weiller, Markus; Horling, Franziska; Mayrhofer, Josef; Schuster, Maria; Falkner, Falko G; Gil-Farina, Irene; Klugmann, Matthias; Scheiflinger, Friedrich; Hoellriegl, Werner; Rottensteiner, Hanspeter
发表日期：2025
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Evidence of protective effects of recombinant ADAMTS13 in a humanized model of sickle cell disease

重组ADAMTS13在人源化镰状细胞病模型中的保护作用证据

期刊:Haematologica
影响因子:7.9
doi:10.3324/haematol.2021.280233
Rossato, Paolo; Federti, Enrica; Matte, Alessandro; Glantschnig, Helmut; Canneva, Fabio; Schuster, Maria; Coulibaly, Sogue; Schrenk, Gerald; Voelkel, Dirk; Dockal, Michael; Plaimauer, Barbara; Andolfo, Immacolata; Iolascon, Achille; Rottensteiner, Hanspeter; Gritsch, Herbert; Scheiflinger, Friedrich; Hoellriegl, Werner; Franceschi, Lucia De
细胞生物学
ADA
发表日期：2022
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Absence of exaggerated pharmacology by recombinant ADAMTS13 in the rat and monkey

重组ADAMTS13在大鼠和猴子中未表现出过度药理作用

期刊:Blood Coagulation & Fibrinolysis
影响因子:1.1
doi:10.1097/MBC.0000000000001064
Rossato, Paolo; Glantschnig, Helmut; Leidenmühler, Peter; Kopic, Alexandra; Ruthsatz, Tanja; Majer, Bernhard; Schuster, Maria; Scheiflinger, Friedrich; Höllriegl, Werner
Rat
ADA
发表日期：2022
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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

BAX 335 B型血友病基因治疗临床试验结果：CpG序列对基因表达的潜在影响

期刊:Blood
影响因子:23.1
doi:10.1182/blood.2019004625
Konkle, Barbara A; Walsh, Christopher E; Escobar, Miguel A; Josephson, Neil C; Young, Guy; von Drygalski, Annette; McPhee, Scott W J; Samulski, R Jude; Bilic, Ivan; de la Rosa, Maurus; Reipert, Birgit M; Rottensteiner, Hanspeter; Scheiflinger, Friedrich; Chapin, John C; Ewenstein, Bruce; Monahan, Paul E
基因治疗
BAX
发表日期：2021
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Blockade of the costimulatory CD28-B7 family signal axis enables repeated application of AAV8 gene vectors

阻断共刺激CD28-B7家族信号轴可实现AAV8基因载体的重复应用

期刊:Journal of Thrombosis and Haemostasis
影响因子:5
doi:10.1111/jth.14757
Frentsch, Marco; Japp, Alberto Sada; Dingeldey, Manuela; Matzmohr, Nadine; Thiel, Andreas; Scheiflinger, Friedrich; Reipert, Birgit M; de la Rosa, Maurus
信号转导
CD2
CD28
发表日期：2020
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Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

开发适用于临床产品上市的AAV8血友病B基因治疗载体的体外生物活性检测方法

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2020.03.013
Lengler, Johannes; Coulibaly, Sogue; Gruber, Bernadette; Ilk, Reinhard; Mayrhofer, Josef; Scheiflinger, Friedrich; Hoellriegl, Werner; Falkner, Falko G; Rottensteiner, Hanspeter
基因治疗
发表日期：2020
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Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

最小必需人凝血因子VIII改变可增强分泌和基因治疗效率

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2020.10.013
Cao, Wenjing; Dong, Biao; Horling, Franziska; Firrman, Jenni A; Lengler, Johannes; Klugmann, Matthias; de la Rosa, Maurus; Wu, Wenman; Wang, Qizhao; Wei, Hongying; Moore, Andrea R; Roberts, Sean A; Booth, Carmen J; Hoellriegl, Werner; Li, Dong; Konkle, Barbara; Miao, Carol; Reipert, Birgit M; Scheiflinger, Friedrich; Rottensteiner, Hanspeter; Xiao, Weidong
Human
基因治疗
发表日期：2020
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Temperature-dependent irreversible conformational change of recombinant ADAMTS13 upon metal ion chelation

金属离子螯合后重组ADAMTS13的温度依赖性不可逆构象变化

期刊:Journal of Thrombosis and Haemostasis
影响因子:5
doi:10.1111/jth.14440
Rottensteiner, Hanspeter; Kaufmann, Stefan; Rathgeb, Anna; Kink, Barbara; Plaimauer, Barbara; Matthiessen, Peter; Hann, Stephan; Scheiflinger, Friedrich
ADA
发表日期：2019
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Identification of cysteine thiol-based linkages in ADAMTS13 in support of a non-proteolytic regulation of von Willebrand factor.

在 ADAMTS13 中鉴定出半胱氨酸硫醇基连接，支持对血管性血友病因子进行非蛋白水解调节

期刊:Journal of Thrombosis and Haemostasis
影响因子:5
doi:10.1111/jth.14602
Rottensteiner Hanspeter, Seyfried Birgit K, Kaufmann Stefan, Fiedler Christian, Dong Jing-Fei, Zheng X Long, Plaimauer Barbara, Scheiflinger Friedrich
心血管
发表日期：2019
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How Full-Length FVIII Benefits from Its Heterogeneity - Insights into the Role of the B-Domain

全长FVIII如何从其异质性中获益——B结构域作用机制研究

期刊:Pharmaceutical Research
影响因子:4.3
doi:10.1007/s11095-019-2599-2
Anzengruber, Julia; Feichtinger, Martin; Bärnthaler, Philipp; Haider, Norbert; Ilas, Josenato; Pruckner, Nina; Benamara, Karima; Scheiflinger, Friedrich; Reipert, Birgit M; Malisauskas, Mantas
发表日期：2019
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