Results 61 to 70 of about 22,643,381 (355)

A hemophilia A mouse model for the in vivo assessment of Emicizumab function.

Blood, 2020
The bispecific antibody Emicizumab is increasingly used for hemophilia A-treatment. However, its specificity for human factors IX and X (FIX, FX) has limited its in vivo functional analysis to primate models of acquired hemophilia.
Stephen Ferrière, I. Peyron, O. Christophe, C. Kawecki, C. Casari, Vincent Muczynski, A. Nathwani, A. Kauskot, P. Lenting, C. Denis   +9 more
semanticscholar   +1 more source

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 more
core   +1 more source

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Journal of Medical Economics, 2020
Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic pain over time.
Keziah Cook, Shaun P. Forbes, K. Adamski, Janice J Ma, A. Chawla, L. Garrison   +5 more
semanticscholar   +1 more source

Spontaneous bleeding in a patient with malignant lymphoma: A case of acquired hemophilia [PDF]

, 2000
Background: Acquired hemophilia is a rare condition which can be associated with lymphoproliferative disease. Case Report: Eleven yea rs after the diagnosis of immunocytoma had been made, a 72-year-old man developed a high-titer factor VIII inhibitor. At
Brack, N., Geiersberger, U., Gesierich, Wolfgang, Hartenstein, R., Munker, Reinhold, Pohlmann, H.   +5 more
core   +1 more source

Hemophilia A and B: molecular and clinical similarities and differences

Haematologica, 2019
Several lines of evidence seem to suggest that the clinical features of hemophilia A and B patients may differ in several points. Genetic background shows a prevalence of missense mutations in hemophilia B compared to the high percentage of mutations ...
G. Castaman, D. Matino
semanticscholar   +1 more source

Recent Advances in mRNA Delivery Systems for Cancer Therapy

Advanced Science, EarlyView.
This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang, Ya‐Nan Fan, Si‐Qi Jiang, Ya‐Jing Ma, Yao‐Ru Yu, Yu‐Xin Qing, Qian‐Ru Li, Yi‐Lin Liu, Song Shen, Jun Wang   +9 more
wiley   +1 more source

Advances on Albumin‐Based Carriers for Anticancer Drug Delivery

Advanced NanoBiomed Research, EarlyView.
This study systematically classifies the drug delivery modes of albumin by examining the characteristics of its surface‐active groups and internal binding sites. It provides an in‐depth analysis of various drug delivery strategies and their corresponding targeting mechanisms.
Ruoli Zhou, Rongbin Zhong, Zhonghui Luo, Hua Wei, Cui‐Yun Yu   +4 more
wiley   +1 more source

Effect of pulsed electromagnetic fields on clinical signs and quality of life in patients with hemophilic arthropathy of the knee joint: A randomized controlled trial

Advanced Biomedical Research, 2020
Background: Hemophilic arthropathy (HA) causes severe joint damage and impairs the quality of life (QoL) of hemophiliacs. This study was undertaken to evaluate the effect of pulsed electromagnetic fields (PEMFs) on the clinical signs and QoL of patients ...
Azam Khami, Mohammad Mohsen Roostayi, Behrouz Parhampour, Zahra Heidari, Hamzeh Baharlouei, Hamid Hoorfar   +5 more
doaj   +1 more source

Techno‐Economic Analysis of Membrane‐Based Purification Platforms for AAV Vector Production

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Technologies for large‐scale manufacturing of viral vectors for gene therapies, such as tangential flow filtration and membrane chromatography, are under development. In these early stages of process development, techno‐economic analyses are useful for identifying membrane properties yielding the greatest impact on process performance. In this
Juan J. Romero, Eleanor W. Jenkins, Jacob I. Monroe, S. Ranil Wickramasinghe, Xianghong Qian, Dibakar Bhattacharyya, Scott M. Husson   +6 more
wiley   +1 more source

ORAL TREATMENT OF HEMOPHILIA [PDF]

, 2007
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome.
Alpan, Oral, Kamala, Tirumalai, Matzinger, Polly, Velander, William Hugold   +3 more
core   +1 more source