Results 91 to 100 of about 16,303 (230)

Full-length FVIII can be expressed at levels as high as BDD FVIII.

, 2014
(A, B) Total mRNAs were extracted from 293T cells (A) or human iPS cells (B), and the level of expression was assessed by quantitative RT-PCR. Expression values were normalized to the level of GAPDH mRNA.
Shinya Yamanaka (202678), Naoko Fujimoto (616381), Noriko Sasakawa (616382), Yasuhide Ohinata (27976), Mitsuhiko Sugimoto (497878), Akitsu Hotta (43917), Hideto Matsui (497881), Midori Shima (497880)   +7 more
core   +1 more source

Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial

Therapeutic Advances in Hematology
Background: Hemophilia A is caused by coagulation factor VIII (FVIII) deficiency and increases bleeding risk during invasive procedures. Objectives: To investigate FVIII concentrate use and bleeding outcomes for invasive procedures after valoctocogene ...
Doris V. Quon, Jiaan-Der Wang, Michael Wang, Dominic Pepperell, Young-Shil Park, Gili Kenet, Johnny Mahlangu, Teh-Liane Khoo, Tara M. Robinson, Konstantia-Maria Chavele, Steven W. Pipe   +10 more
doaj   +1 more source

Pharmacokinetic Studies of Factor VIII in Chinese Boys with Severe Hemophilia A: A Single-Center Study

Chinese Medical Journal, 2018
Background: Although much attention has been paid to the pharmacokinetics (PKs) of different factor VIII (FVIII) concentrates in persons with hemophilia A (HA), limited information is available in young boys with severe HA.
Zhen-Ping Chen, Pei-Jing Li, Gang Li, Ling Tang, Ying-Zi Zhen, Xin-Yi Wu, Xiao-Ling Cheng, Koon Hung Luke, Victor S Blanchette, Man-Chiu Poon, Qiu-Lan Ding, Run-Hui Wu   +11 more
doaj   +1 more source

Changes in Hypertrophic Synovium Status in Adult Patients With Severe Hemophilia A Receiving Emicizumab Prophylaxis: The RESOLVE Study

Haemophilia, EarlyView.
ABSTRACT Background Evidence on the impact of emicizumab on joint health, particularly hypertrophic synovium (HS), remains limited. Objectives To evaluate changes in HS, using point‐of‐care ultrasound and clinical examination in People with Haemophilia (PwH) switching from replacement therapy to emicizumab.
Matteo Nicola Dario Di Minno, Erminia Baldacci, Ilenia Lorenza Calcaterra, Guido D'Errico, Cristina Santoro, Alessandra Strangio, Silvia Sorella, Marzia Leotta, Rita Carlotta Santoro   +8 more
wiley   +1 more source

Patient Perspectives in Haemophilia A: Unmet Needs of Individuals Receiving Factor and Non‐Factor Treatments

Haemophilia, EarlyView.
ABSTRACT Introduction There is limited qualitative published real‐world data on treatment patterns among people with severe haemophilia A (PwSHA), particularly those with unmet needs. Aim This qualitative study aimed to explore the impact of taking prophylactic extended half‐life factor VIII (EHL‐FVIII) or emicizumab on psychosocial and physical ...
Nana Kragh, Veruska Carboni, Duygu Bozkaya, Idaira Rodriguez‐Santana, Claudia Mighiu, Bethany Franks, Arnoud Plat, Randall Curtis   +7 more
wiley   +1 more source

Immuntoleranzinduktion mit hoch dosiertem FVIII und intravenösen Immunglobulin-Pulsen

, 2010
SummaryThe development of neutralizing allo-antibodies against factor VIII (FVIII) or FVIII inhibitors is a severe complication in the treatment of haemophilia A. About 25% of the children with severe haemophilia A develop FVIII inhibitors.
C. Pinkwart, M. Stiefel, R. Haase, D. Forsberg, N. Merkel, C. Mauz-Körholz   +5 more
core   +1 more source

Physical Activity, Bleeding Incidence and Health‐Related Quality of Life, in Patients With Haemophilia A Without Inhibitors, Treated With Emicizumab: A Post‐Hoc Descriptive Analysis of the Power Study Data

Haemophilia, EarlyView.
Matteo Nicola Dario Di Minno, Berardino Pollio, Ezio Zanon, Gaetano Giuffrida, Sara Bendinelli, Raffaella Perrone, Giancarlo Castaman, the POWER study sites group   +7 more
wiley   +1 more source

Advanced Molecular Analysis in Hemophilia A in a Single Step: Next Generation Sequencing (NGS) and Copy Number Variation (CNV) Analysis

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Background Hemophilia A, an X‐linked bleeding disorder caused by pathogenic variants in the F8 gene, requires precise genetic diagnosis for optimal management. Conventional stepwise sequence and copy number variation (CNV) analyses are time‐consuming and may leave some cases unresolved.
Enise Avci Durmusalioglu, Esra Isik, Basak Durmus Ozen, Mehmet Mert Topaloglu, Nihal Karadas, Kaan Kavakli, Melike Sezgin Evim, Tolga Aslan, Erşen Kavak, Adalet Meral Gunes, Funda Tayfun Kupesiz, Alphan Kupesiz, Fahri Sahin, Tahir Atik   +13 more
wiley   +1 more source

Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A

, 2010
The natural cell type(s) that synthesize and release factor VIII (FVIII) into the circulation are still not known with certainty. In vitro studies indicate that artificial expression of FVIII in endothelial cells produces an intracellular pool of FVIII ...
Erin L. Kuether, Robert R. Montgomery, Brian C. Cooley, Hartmut Weiler, Qizhen Shi, Scot A. Fahs   +5 more
core   +1 more source

Factor VIII bypasses CD91/LRP for endocytosis by dendritic cells leading to T-cell activation

Haematologica, 2008
Background The development of factor VIII (FVIII) inhibitors remains the major hurdle in the clinical management of patients with hemophilia A. FVIII uptake by professional antigen-presenting cells (APC) is the first step involved in initiation of immune
Suryasarathi Dasgupta, Ana Maria Navarrete, Sebastien André, Bharath Wootla, Sandrine Delignat, Yohann Repessé, Jagadeesh Bayry, Antonino Nicoletti, Evgueni L. Saenko, Roseline d’Oiron, Marc Jacquemin, Jean-Marie Saint-Remy, Srini V. Kaveri, Sebastien Lacroix-Desmazes   +13 more
doaj   +1 more source