Results 61 to 70 of about 113,823 (359)

Effects of different storage temperatures and durations on the activity of coagulation factor Ⅷ and Ⅸ in whole blood

Zhongguo shuxue zazhi
[Objective] To investigate the effects of different storage temperatures and durations on the activities of coagulation factor Ⅷ (Factor Ⅷ, FⅧ) and coagulation factor Ⅸ (Factor Ⅸ, FⅨ) after whole blood collection, so as to provide data support for the ...
WANG Hehe, WANG Tiantian, WANG Jie, QIAO Cuicui, LIU Wei, ZHANG Xueqin, CHENG Yan, FANG Yunhai, ZHANG Xinsheng   +8 more
doaj   +1 more source

A multicenter, open-label, phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.

Blood, 2019
Emicizumab, a bispecific humanized monoclonal antibody, bridges activated factor (F) IX and FX to restore the function of missing activated FVIII in hemophilia A.
G. Young, R. Liesner, Tiffany Y. Chang, R. Sidonio, J. Oldenburg, V. Jiménez‐Yuste, J. Mahlangu, R. Kruse-Jarres, Michael Wang, Marianne Uguen, M. Doral, Lilyan Y. Wright, C. Schmitt, G. Levy, M. Shima, M. Mancuso   +15 more
semanticscholar   +1 more source

Occurrence of FVIII Inhibitors in Hemophilia A Patients Following an Institutional Switch to a Third Generation B-Domain-Deleted FVIII

Clinical and Applied Thrombosis/Hemostasis, 2023
In 2018, Refacto AF R , a B-domain-deleted third-generation FVIII concentrate, became our preferential product. After the introduction, the development of inhibitors was prospectively monitored; retrospectively, we sought for risk factors in the patients
Louise H Hooimeijer MD, Marjet A Stein-Wit MD, Marja AJ Voskuilen RN, Michaël V Lukens PhD, Karina Meijer MD, PhD, Anja BU Mäkelburg MD, PhD, Rienk YJ Tamminga MD, PhD   +6 more
doaj   +1 more source

Incidence and mortality rates of intracranial hemorrhage in hemophilia: a systematic review and meta-analysis.

Blood, 2021
Intracranial hemorrhage (ICH) is a severe complication that is relatively common among hemophilia patients. This systematic review aimed to obtain more precise estimates of ICH incidence and mortality in hemophilia, which may be important for patients ...
A. Zwagemaker, S. Gouw, Julie J Jansen, C. Vuong, M. Coppens, Q. Hu, Xiao-qin Feng, S. Kim, J. G. van der Bom, K. Fijnvandraat   +9 more
semanticscholar   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]

, 2018
Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana, Gao, Kewa, Kumar, Priyadarsini, Lankford, Lee, Pivetti, Christopher, Wang, Aijun, Wang, Chuwang   +6 more
core   +1 more source

Gene Therapy in Hemophilia: Recent Advances

International Journal of Molecular Sciences, 2021
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors.
E. Rodríguez‐Merchán, J. A. De Pablo-Moreno, A. Liras   +2 more
semanticscholar   +1 more source

Dual‐Functional Polyphosphoesters for Gene Delivery: Synergistic Effects of Guanidinium and Hydrophobic Side Chains in Degradable Polymers

Advanced Healthcare Materials, EarlyView.
This study presents guanidinium‐ and indole‐functionalized polyphosphoesters as degradable, non‐viral gene delivery vectors. Through precise tuning of charge density and hydrophobicity, these polymers form stable polyplexes with low toxicity. Remarkably, minor structural changes yield up to 200‐fold differences in transfection efficiency, highlighting ...
Markus Kötzsche, Andreas Dzierza, Jan Egger, Timo Rheinberger, Christin Weilandt, Frederik R. Wurm, Ivo Nischang, Dagmar Fischer, Kalina Peneva   +8 more
wiley   +1 more source

Retrospective analysis of clinical data from 171 low-income patients with hemophilia in Shandong Province

Scientific Reports
To enhance attention on low-income patients with hemophilia, it is essential to urge governments and coagulation factor manufacturers to increase their investment in hemophilia care.
Jie Wang, Yan Cheng, Yunhai Fang, Cuicui Qiao, Tiantian Wang, Hehe Wang, Xueqin Zhang, Qiang Li   +7 more
doaj   +1 more source