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56 Newest Publications about the topic hemophilia

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Pharmacokinetics, tissue distribution, excretion, and metabolite profiling of PEGylated rFIX (nonacog beta pegol, N9-GP) in rats

14-Aug-2016 | Author(s): Ola Sternebring, Jesper Kammersgaard Christensen, Inga Bjørnsdottir, European Journal of Pharmaceutical Science, 2016

Publication date: 20 September 2016 Source:European Journal of Pharmaceutical Sciences, Volume 92 Author(s): Ola Sternebring, Jesper Kammersgaard Christensen, Inga Bjørnsdottir Nonacog beta pegol (N9-GP) is a novel recombinant factor IX conjugated with a 40-kDa branched polyethylene ...

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Factor VIII associated with lipidic nanoparticles retains efficacy in the presence of anti‐Factor VIII antibodies in Hemophilia A mice

15-Jul-2016 | Krithika A. Shetty, Matthew P. Kosloski, Donald E. Mager, Sathy V. Balu‐Iyer, Biopharmaceutics & Drug Disposition, 2016

Abstract Development of inhibitory antibodies against Factor VIII (FVIII) is a major challenge in Hemophilia A (HA) therapy. Such antibodies develop in nearly 30% of the patients receiving replacement FVIII, abrogating therapeutic efficacy. In this work, we evaluated whether B‐domain deleted ...

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[Policy Forum] Paying for future success in gene therapy

27-May-2016 | Stuart H. Orkin; Philip Reilly, Science , 2016

Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited ...

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Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success

04-Apr-2016 | Tuan Huy Nguyen, Ignacio Anegon, EMBO Molecular Medicine, 2016

Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B. In hemophilia, reconstitution with a mere 1–2% of the clotting factor improves the quality of life, ...

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CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

10-Mar-2016 | Yuting Guan, Yanlin Ma, Qi Li, Zhenliang Sun, Lie Ma, Lijuan Wu, Liren Wang, Li Zeng, Yanjiao Shao, Yuting Chen, Nin ..., EMBO Molecular Medicine, 2016

Abstract The X‐linked genetic bleeding disorder caused by deficiency of coagulator factor IX, hemophilia B, is a disease ideally suited for gene therapy with genome editing technology. Here, we identify a family with hemophilia B carrying a novel mutation, Y371D, in the human F9 gene. The ...

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Y‐chromosome identification in circulating cell‐free fetal DNA using surface plasmon resonance

06-Feb-2016 | Giulia Breveglieri, Elisabetta Bassi, Silvia Carlassara, Lucia Carmela Cosenza, Patrizia Pellegatti, Giovanni Guerra ..., Prenatal Diagnosis, 2016

Abstract Objective Since the discovery of cell‐free fetal DNA (cffDNA) in maternal plasma, diagnostic non‐invasive prenatal methods have been developed or optimized for fetal sex determination and identification of genetic diseases. As far as fetal sex determination, this might be important ...

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Synergistic inhibition of PARP‐1 and NF‐κB signaling down‐regulates immune response against recombinant AAV2 vectors during hepatic gene therapy

07-Oct-2015 | Sangeetha Hareendran, Banumathi Ramakrishna, Giridhara R. Jayandharan, European Journal of Immunology, 2015

Host immune response remains a key obstacle to widespread application of AAV based gene therapy. Thus, targeted inhibition of the signaling pathways that trigger such immune responses will be beneficial. Previous studies have reported that DNA damage response proteins such as PARP‐1 negatively ...

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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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