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27 Newest Publications about the topic hemophiliarss
16-05-2013 | Gouri Shankar Pandey et al., PLoS Computational Biology, 2013
by Gouri Shankar Pandey, Chen Yanover, Tom E. Howard, Zuben E. Sauna The development of neutralizing anti-drug-antibodies to the Factor VIII protein-therapeutic is currently the most significant impediment to the effective management of hemophilia A. Common non-synonymous single nucleotide ...
01-05-2013 | Jérôme Lane; Paul J. McLaren; Lucy Dorrell; Kevin V. Shianna; Amanda Stemke; Kimberly Pelak; Stephen Moore; Johannes ..., Human Molecular Genetics, 2013
Human genetic variation contributes to differences in susceptibility to HIV-1 infection. To search for novel host resistance factors, we performed a genome-wide association study (GWAS) in hemophilia patients highly exposed to potentially contaminated factor VIII infusions. Individuals with ...
16-04-2013 | João Paulo Pimentel, Daniel Gonçalves Chaves, Ana Ruth Silva Araújo, Erbênia Maria Martins de Araújo, Liziara da Sil ..., Journal of Medical Virology, 2013
Abstract In the past decades patients with hemophilia were infected commonly by hepatitis C virus (HCV) and a significant number of patients are infected chronically. Focusing on the role of the immune system for controlling and or maintaining HCV infection, the leukocyte and cytokine profiles ...
06-02-2013 | Alisa Opar, Nature Medicine, 2013
Nature Medicine 19, 121 (2013). doi:10.1038/nm0213-121 Author: Alisa Opar
17-01-2013 | Svetla Stoilova‐McPhie, Gillian C. Lynch, Steven Ludtke, B. Montgomery Pettitt, Biopolymers, 2013
Abstract Factor VIII (FVIII) is the blood coagulation protein when defective or deficient causes for hemophilia A, a severe hereditary bleeding disorder. Activated FVIII (FVIIIa) is the co‐factor to the serine protease Factor IXa (FIXa) within the membrane‐bound Tenase complex, responsible for ...
01-01-2013 | Mark W Skinner, Molecular Therapy, 2013
Gene Therapy for Hemophilia: Addressing the Coming Challenges of Affordability and Accessibility Molecular Therapy 21, 1 (January 2013). doi:10.1038/mt.2012.272 Author: Mark W Skinner
26-12-2012 | Amanda B. Payne, Connie H. Miller, Fiona M. Kelly, J. Michael Soucie, W. Craig Hooper, Human Mutation, 2012
ABSTRACT Genotyping efforts in hemophilia A (HA) populations in many countries have identified large numbers of unique mutations in the Factor VIII gene (F8). To assist HA researchers conducting genotyping analyses, we have developed a listing of F8 mutations including those listed in existing ...
10-12-2012 | Jacobs, Frank ; Gordts, Stephanie C.; Muthuramu, Ilayaraja ; De Geest, Bart, Pharmaceuticals, 2012
The liver is a target for gene therapy of inborn errors of metabolism, of hemophilia, and of acquired diseases such as liver cancer and hepatitis. The ideal gene transfer strategy should deliver the transgene DNA to parenchymal liver cells with accuracy and precision in the absence of side ...
09-11-2012 | Philip M. Zakas et al., PLoS ONE, 2012
by Philip M. Zakas, Bagirath Gangadharan, Graca Almeida-Porada, Christopher D. Porada, H. Trent Spencer, Christopher B. Doering Animal models of the bleeding disorder, hemophilia A, have been an integral component of the biopharmaceutical development process and have facilitated the ...
01-07-2012 | Alan R. Rushton, Journal of the History of Medicine and Allied Sciences, 2012
Hemophilia is a rare bleeding disorder inherited by males born of unaffected female carriers of the trait. British physicians became knowledgeable about this hereditary disease early in the nineteenth century as they investigated families transmitting the character through several generations. ...