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60 Newest Publications about the topic hemophilia

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Seroprevalence and clinical characteristics of viral hepatitis in transfusion-dependent thalassemia and hemophilia patients

09-Jun-2017 | Tyng-Yuan Jang; Pei-Chin Lin; Ching-I Huang; Yu-Mei Liao; Ming-Lun Yeh; Yu-Sheng Zeng; Po-Cheng Liang; Wan-Yi Hsu; S ..., PLoS ONE, 2017

by Tyng-Yuan Jang, Pei-Chin Lin, Ching-I Huang, Yu-Mei Liao, Ming-Lun Yeh, Yu-Sheng Zeng, Po-Cheng Liang, Wan-Yi Hsu, Shih-Pien Tsai, Zu-Yau Lin, Shinn-Cherng Chen, Jee-Fu Huang, Chia-Yen Dai, Chung-Feng Huang, Shyh-Shin Chiou, Wan-Long Chuang, Ming-Lung Yu Background/Aims Transfusion dependent ...

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Systemic delivery of factor IX messenger RNA for protein replacement therapy [Medical Sciences]

07-Mar-2017 | Suvasini Ramaswamy; Nina Tonnu; Kiyoshi Tachikawa; Pattraranee Limphong; Jerel B. Vega; Priya P. Karmali; Pad Chivuk ..., Proceedings of the National Academy of Sciences current issue, 2017

Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (FIX)-deficient mouse model of hemophilia ...

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The Utilization of Rehabilitation in Patients with Hemophilia A in Taiwan: A Nationwide Population-Based Study

30-Sep-2016 | Chien-Min Chen; Yao-Hsu Yang; Chia-Hao Chang; Chih-Cheng Chen; Pau-Chung Chen, PLoS ONE, 2016

by Chien-Min Chen, Yao-Hsu Yang, Chia-Hao Chang, Chih-Cheng Chen, Pau-Chung Chen Introduction Rehabilitation plays an important role in the physical health of patients with hemophilia. However, comprehensive information regarding the utilization of rehabilitation for such patients remains ...

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A Cure For Hemophilia: the Promise Becomes a Reality

01-Sep-2016 | Roland W Herzog, Molecular Therapy, 2016

A Cure For Hemophilia: the Promise Becomes a Reality Molecular Therapy 24, 1503 (september 2016). doi:10.1038/mt.2016.169 Author: Roland W Herzog

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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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