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49 Newest Publications about the topic hemophilia

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Regulation of a strong F9 cryptic 5′ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides

01-Sep-2015 | Dario Balestra; Elena Barbon; Daniela Scalet; Nicola Cavallari; Daniela Perrone; Silvia Zanibellato; Francesco Berna ..., Human Molecular Genetics, 2015

Mutations affecting specific splicing regulatory elements offer suitable models to better understand their interplay and to devise therapeutic strategies. Here we characterize a meaningful splicing model in which numerous Hemophilia B-causing mutations, either missense or at the donor splice site ...

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Personalized approaches to the treatment of hemophilia A and B

03-Aug-2015 | Vijaya L Simhadri; Aditi Sengupta Banerjee; Jonathan Simon; Chava Kimchi-Sarfaty; Zuben E Sauna, Personalized Medicine, 2015

The recognition that individuals respond differently to the same medication is not new and dates almost to the founding of western medicine. In the last century it came to be recognized that genetic factors influence the heterogeneity of individual responses to medications with respect to both ...

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Personalized approaches to the treatment of hemophilia A and B

03-Aug-2015 | Vijaya L Simhadri; Aditi Sengupta Banerjee; Jonathan Simon; Chava Kimchi-Sarfaty; Zuben E Sauna, Personalized Medicine, 2015

The recognition that individuals respond differently to the same medication is not new and dates almost to the founding of western medicine. In the last century it came to be recognized that genetic factors influence the heterogeneity of individual responses to medications with respect to both ...

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Exposure of FVIII in the Presence of Phosphatidyl Serine Reduces Generation of Memory B‐Cells and Induces Regulatory T‐Cell‐Mediated Hyporesponsiveness in Hemophilia A Mice

03-Jun-2015 | Radha Ramakrishnan, Andrew Davidowitz, Sathy V. Balu‐Iyer, Journal of Pharmaceutical Sciences, 2015

A major complication of replacement therapy with Factor VIII (FVIII) for hemophilia A (HA) is the development of unwanted immune responses. Previous studies showed that administration of FVIII in the presence of phosphatidyl serine (PS) reduced the development of anti‐FVIII antibodies in HA ...

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Turoctocog alfa for hemophilia A

23-Mar-2015 | Drug Design, Development and Therapy, 2015

Takedani H, Hirose J

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Intravenous administration of Factor VIII–O-Phospho-L-Serine (OPLS) complex reduces immunogenicity and preserves pharmacokinetics of the therapeutic protein

26-Oct-2014 | Author(s): Puneet Gaitonde , Vivek S. Purohit , Sathy V. Balu-Iyer, European Journal of Pharmaceutical Science, 2014

Publication date: Available online 20 October 2014 Source:European Journal of Pharmaceutical Sciences Author(s): Puneet Gaitonde , Vivek S. Purohit , Sathy V. Balu-Iyer Hemophilia A is a bleeding disorder caused by the deficiency of an important coagulation factor; Factor VIII (FVIII). ...

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The extraordinary career of Professor Dr. Simon van Creveld

30-Sep-2014 | Paul J. W. Stoelinga, Walter E. Berdon, M. Michael Cohen, American Journal of Medical Genetics Part A, 2014

Simon van Creveld received both the MD and PhD degrees and had a multifaceted medical and scientific education at many hospitals and research institutes in the Netherlands, Germany, and the UK. He and his wife were the first to develop insulin for the Netherlands. His major interests were in ...

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O‐Phospho‐l‐Serine mediates Hyporesponsiveness toward FVIII in Hemophilia A‐Murine Model by Inducing Tolerogenic Properties in Dendritic Cells

30-Sep-2014 | Anas M. Fathallah, Radha Ramakrishnan, Sathy V. Balu‐Iyer, Journal of Pharmaceutical Sciences, 2014

The clinical use of therapeutic proteins can be complicated by the development of anti‐product antibodies. We have previously observed that O‐phospho‐l‐serine (OPLS) reduced antibody response to FVIII in Hemophilia‐A (HA) mice. However, the mechanism underlying this observation is not clear. We ...

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Delivery of Full-Length Factor VIII Using a piggyBac Transposon Vector to Correct a Mouse Model of Hemophilia A

15-Aug-2014 | Hideto Matsui et al., PLoS ONE, 2014

by Hideto Matsui, Naoko Fujimoto, Noriko Sasakawa, Yasuhide Ohinata, Midori Shima, Shinya Yamanaka, Mitsuhiko Sugimoto, Akitsu Hotta Viral vectors have been used for hemophilia A gene therapy. However, due to its large size, full-length Factor VIII (FVIII) cDNA has not been successfully ...

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The prevalence of neutralizing antibodies against adeno‐associated virus capsids is reduced in young Japanese individuals

17-Oct-2013 | Jun Mimuro, Hiroaki Mizukami, Midori Shima, Tadashi Matsushita, Masashi Taki, Shinji Muto, Satoshi Higasa, Michio Sa ..., Journal of Medical Virology, 2013

Abstract Pre‐existing antibodies against adeno‐associated virus (AAV), caused by natural AAV infections, interfere with recombinant AAV vector‐mediated gene transfer. We studied the prevalence of neutralizing antibodies against AAV serotypes 1, 2, 5, 8, and 9 in healthy subjects (n = 85) and ...

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